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Endo-Porter simple delivery reagent
Citations of papers using Endo-Porter are here.
  1. What is it and how does it work?
  2. Endo-Porter is a novel peptide* explicitly designed to do what all existing peptide or lipid-based delivery reagents can not: deliver substances into the cytosol of cells by an endocytosis-mediated process that avoids damaging the plasma membrane of the cell. This mechanism prevents the loss of vital cell contents with its associated toxicity. Endo-Porter requires no interaction with the cargo to be delivered and allows the cargo that is endocytosed along with Endo-Porter to exit the endosome and enter the cytosol. Detailed studies with a FITC-anti-actin antibody fragment (Sigma F-3046) and fluorescent high-molecular weight dextrans suggest Endo-Porter can deliver high concentrations of cargo exceeding 70kD into both adherent and non-adherent cells. Unlike the many delivery reagents that claim effectiveness in the presence of serum, Endo-Porter actually works with serum concentrations up to the 10% standard. Endo-Porter is excellent at delivering peptides, proteins, and Morpholino antisense oligos as well! We do not recommend using Endo-Porter for delivery of large (>100 Kd) proteins, as efficacy is poor.

    siRNA delivery: Endo-Porter has been reported effective for delivering siRNA in HeLa, Huh-7 and Hek293T cultures [Bartz et al. 2011]. Combined with poly-lysine, Endo-Porter delivered siRNA efficiently in rat hepatoma H4IIEC3 cells [Inoue et al. 2008]. Endo-Porter has been reported to deliver siRNA in vivo when encapsulated in glucan shells [Tesz et al. 2011, Aouadi et al. 2009]. Our tests have not found Endo-Porter effective for delivery of plasmid DNA.

    * Patented: US 7,084,248 B2

    Figure: Endo-Porter delivery mechanism and image of Endo-Porter delivery of 70 kD dextran tagged with Texas Red

    View or download an animation of Endo-Porter's mechanism of action by selecting one of the following two choices:

    Windows Media Endo-Porter animation
    Apple QuickTime Endo-Porter animation

    Animation by Pathworks of Corvallis, OR.

  3. What are the advantages of Endo-Porter over other delivery reagents?
  4. Most other delivery reagents require pre-incubation or optimized reagent-to-cargo ratio in order to achieve delivery, but not Endo-Porter. You can add as much or as little cargo as desired and use the same amount of Endo-Porter EVERY time. Endo-Porter lasts indefinitely and can be stored at room temperature or below.

  5. What do I get and how much does it cost?
  6. You will receive 1.0 mL of 1.0 mM Endo-Porter in solution. This is enough to deliver up to about 150 mL of cells with Endo-Porter (optimal dose varies with the cell type). Endo-Porter can be ordered online .

    We recommend Endo-Porter PEG for all new users, as this is our highest efficacy formulation with even less toxicity than the previous-best DMSO formulation. Our legacy formulations are still available for replicating previous experiments.

    The available formulations are:
    1. Endo-Porter PEG (preferred), 1 mM Endo-Porter in 10% polyethylene glycol 1500 MW, aqueous.
    2. Endo-Porter DMSO (legacy), 1 mM Endo-Porter in DMSO.
    3. Endo-Porter Aqueous (legacy), 1 mM Endo-Porter in an aqueous solution of mannitol.
    For the current price of Endo-Porter, please see out price list. The price list is linked under the "Ordering" tab of our website.

    For additional questions or assistance from our Ph.D.-level customer support staff use our online live help, submit a question, or call (541) 929-7840 ext. 1.

  7. Endo-Porter excels in independent comparison of nine cytosolic delivery reagents

  8. When delivering Atto488-bovine serum albumin into cultured human sarcoma cells using nine different commercial delivery products, only Endo-Porter achieved clear cytosolic delivery.

    "A primarily endosomal uptake of proteins is consistent with the observation that cytoplasmic signals could only be observed in Endo-Porter treated cells. The Endo-Porter reagent is a specialized reagent to disrupt endosomal membranes as a consequence of endosomal acidification and it is the only one of the reagents tested which has exclusively been invented to overcome endosomal entrapment."[1]

    Endo-Porter also delivered to the largest fraction of cells: "Comparing the different transduction reagents, the percentage of fluorescence positive cells varied between 89.2 (TurboFect) and 96.8% (Endo-Porter)."[1]

    [1] Mellert K, Lamla M, Scheffzek K, Wittig R, Kaufmann D. Enhancing endosomal escape of transduced proteins by photochemical internalisation. PLoS One. 2012;7(12):e52473. doi: 10.1371/journal.pone.0052473. Epub 2012 Dec 21.

  9. The protocol is simple: squirt and swirl.
  10. Our recommended delivery procedure:

    1. Replace spent culture medium with fresh medium (preferably 10% serum).
    2. Add the desired cargo and swirl to mix.
    3. Add 6 µL of Endo-Porter for every 1 mL of culture medium & cargo and immediately swirl to mix.

    In most cases, long-term (72 hour) and short-term (24 hour) delivery is optimal at 6 µM Endo-Porter (6 µL/mL); however, cell lines vary in their response to Endo-Porter and it is prudent to start experiments with a new cell line by doing a ranging study for efficacy and toxicity at 2, 4, and 6 µL/mL Endo-Porter. Check the cells at 72 hours for signs of toxicity, then use the highest concentration that the cells tolerate for subsequent delivery procedures. Endo-Porter PEG may be tolerated at even higher doses. Unlike most delivery solutions, 10% serum concentrations yield better results than reduced serum levels. Additional cargo and Endo-Porter can be added with each media replacement if desired. For delivery of Morpholino oligos, we suggest you start with 10 µM oligo final concentration in the culture medium then adjust concentrations as needed to achieve effective and specific gene knockdown.

    Endo-Porter protocol (PDF)

    Endo-Porter is not for use in humans.
  11. Citations of Endo-Porter experiments

  12. Khan D, Ramachandiran I, Vasu K, China A, Khan K, Cumbo F, Halawani D, Terenzi F, Zin I, Long B, Costain G, Blaser S, Carnevale A, Gogonea V, Dutta R, Blankenberg D, Yoon G, Fox PL Homozygous EPRS1 missense variant causing hypomyelinating leukodystrophy-15 alters variant-distal mRNA m6A site accessibility Nat Commun. 2024 May 20;15(1):4284. doi: 10.1038/s41467-024-48549-x. PMID: 38769304.

    A description of the development of Endo-Porter has been published:

    Summerton JE. Endo-Porter: A Novel Reagent for Safe, Effective Delivery of Substances into Cells. Ann N Y Acad Sci. 2005 Nov;1058:62-75
    cell culture: NIH/3T3

    Bardhan A, Brown W, Albright S, Tsang M, Davidson L, Deiters A. Direct Activation of Nucleobases with Small Molecules for the Conditional Control of Antisense Function. Angew Chem Int Ed Engl. 2024 Feb 27:e202318773. doi: 10.1002/anie.202318773. Online ahead of print.
    Endo-Porter PEG with HEK293T cultures

    Geist Hauserman J, Laverty CG, Donkervoort S, Hu Y, Silverstein S, Neuhaus SB, Saade D, Vaughn G, Malicki D, Kaur R, Li Y, Luo Y, Liu P, Burr P, Foley AR, Mohassel P, Bönnemann CG. Clinical, immunohistochemical, and genetic characterization of splice-altering biallelic DES variants: Therapeutic implications. HGG Adv. 2024 Feb 2;5(2):100274. doi: 10.1016/j.xhgg.2024.100274. Online ahead of print.

    Doisy M, Vacca O, Fergus C, Gileadi T, Verhaeg M, Saoudi A, Tensorer T, Garcia L, Kelly VP, Montanaro F, Morgan JE, van Putten M , Aartsma-Rus A, Vaillend C, Muntoni F, Goyenvalle A. Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model. Biomedicines. 2023;11(12):3243. doi:10.3390/biomedicines11123243
    H2K-mdx52 myotubes, primary-mdx52 myotubes

    Lui A, Patel RS, Krause-Hauch M, Sparks RP, Patel NA. Regulation of Human Sortilin Alternative Splicing by Glucagon-like Peptide-1 (GLP1) in Adipocytes. Int J Mol Sci. 2023 Sep 20;24(18):14324. doi: 10.3390/ijms241814324.
    cell culture: human diabetic pre-adipocytes

    Gasparski AN, Moissoglu K, Pallikkuth S, Meydan S, Guydosh NR, Mili S. mRNA Location and Translation Rate Determine Protein Targeting to Dual Destinations. bioRxiv. 2023;[preprint] doi:10.1101/2023.04.24.538105
    cell culture: MDA-MB-231

    Preußner M, Smith HL, Hughes D, Zhang M, Emmerichs AK, Scalzitti S, Peretti D, Swinden D, Neumann A, Haltenhof T, Mallucci GR, Heyd F. ASO targeting RBM3 temperature-controlled poison exon splicing prevents neurodegeneration in vivo. EMBO Mol Med. 2023 Mar 22:e17157. doi: 10.15252/emmm.202217157. Online ahead of print.

    Chan SN, Pek JW. Distinct biogenesis pathways may have led to functional divergence of the human and Drosophila Arglu1 sisRNA. EMBO Rep. 2022 Dec 19:e54350. doi: 10.15252/embr.202154350. Online ahead of print.
    cell culture: MDA-MB-231 and HEK293T

    Catasús N, Rosas I, Bonache S, Negro A, Torres-Martin M, Plana-Pla A, Salvador H, Serra E, Blanco I, Castellanos E, on behalf of the NF2-related SWN Spanish National Reference Centre HUGTP-ICO-IGTP. Antisense oligonucleotides targeting exon 11 are able to partially rescue the NF2-related schwannomatosis phenotype in vitro. Mol Ther Nucleic Acids. 2022;[in press] doi:10.1016/j.omtn.2022.10.026.
    cell culture: human patient-derived fibroblasts

    Bisom TC, White LA, Lanchy J-M, Lodmell JS. RIOK3 and Its Alternatively Spliced Isoform Have Disparate Roles in the Innate Immune Response to Rift Valley Fever Virus (MP12) Infection. Viruses. 2022;14(9):2064. doi:10.3390/v14092064
    cell culture: HEK293

    Zhang Y, Siraj MA, Chakraborty P, Tseng R, Ku L-T, Das S, Dey A, Dwivedi SKD, Rao G, Zhang M, Yang D, Hossen MN, Ding W-Q, Fung K-M, Bhattacharya R, Escobar-Hoyos L, Mukherjee P. Activation of ERK by altered RNA splicing in cancer. bioRxiv. 2022;[preprint] doi:10.1101/2022.08.31.505957
    Panc1 & AsPc1 pancreatic cancer lines

    Cripe TP, Hutzen B, Currier MA, Chen CY, Glaspell AM, Sullivan GC, Hurley JM, Deighen MR, Venkataramany AS, Mo X, Stanek JR, Miller AR, Wijeratne S, Magrini V, Mardis ER, Mendell JR, Chandler DS, Wang PY. Leveraging gene therapy to achieve long-term continuous or controllable expression of biotherapeutics. Sci Adv. 2022 Jul 15;8(28):eabm1890. doi: 10.1126/sciadv.abm1890. Epub 2022 Jul 13.
    Endo-Porter and 293T cells

    Liu Y, Yang Y, Lin Y, Wei B, Hu X, Xu L, Zhang W, Lu J. N6 -methyladenosine-modified circRNA RERE modulates osteoarthritis by regulating β-catenin ubiquitination and degradation. Cell Prolif. 2022 Jun 22:e13297. doi: 10.1111/cpr.13297. Online ahead of print.
    Delivery to primary human chondrocytes

    Vorlova S, Blahetek G, Barbieri R, Kerstan Y, Rosa A, Bundalo M, Egg M, Butt E, Henke E, Dölken L, Houben R, Schilling B, Fischer U, Erhard F, Zernecke A. U1 snRNP suppresses microRNA biogenesis by alternative intronic polyadenylation in melanoma. bioRxiv. 2022;[preprint] doi:10.1101/2022.06.01.479622

    Judd M, Place AR. A Strategy for Gene Knockdown in Dinoflagellates. Microorganisms. 2022;10:1131. doi:10.3390/microorganisms10061131
    Endo-Porter delivery of Morpholinos into cultured dinoflagellates

    Tse KM, Vandenbon A, Cui X, Mino T, Uehata T, Yasuda K, Sato A, Tsujimura T, Hia F, Yoshinaga M, Kinoshita M, Okuno T, Takeuchi O. Enhancement of Regnase-1 expression with stem loop-targeting antisense oligonucleotides alleviates inflammatory diseases. Sci Transl Med. 2022 May 11;14(644):eabo2137. doi: 10.1126/scitranslmed.abo2137. Epub 2022 May 11.

    Porpiglia E, Mai T, Kraft P, Holbrook CA, de Morree A, Gonzalez VD, Hilgendorf K, Fresard L, Trejo A, Bhimaraju S, Jackson PK, Fantl WJ, Blau HM. Elevated CD47 is a hallmark of dysfunctional aged muscle stem cells that can be targeted to augment regeneration. bioRxiv. 2022;[preprint] doi:10.1101/2022.04.29.489435
    cell culture: muscle stem cells

    Brooks KE, Daughtry BL, Davis B, Yan MY, Fei SS, Shepherd S, Carbone L, Chavez SL. Molecular contribution to embryonic aneuploidy and karyotypic complexity in initial cleavage divisions of mammalian development. Development. 2022 Apr 1;149(7):dev198341. doi: 10.1242/dev.198341. Epub 2022 Apr 14.
    Endo-Porter delivery to Madin-Darby Bovine Kidney (MDBK) cells

    Siddaway R, Milos S, Vadivel AKA, Dobson THW, Swaminathan J, Ryall S, Pajovic S, Patel PG, Nazarian J, Becher O, Brudno M, Ramani A, Gopalakrishnan V, Hawkins C. Splicing is an alternate oncogenic pathway activation mechanism in glioma. Nat Commun. 2022 Jan 31;13(1):588. doi: 10.1038/s41467-022-28253-4.
    cell culture: U87 GBM, pediatric and adult high-grade diffuse glioma cells

    Leier A, Moore M, Liu H, Daniel M, Hyde AM, Messiaen L, Korf BR, Selvakumaran J, Ciszewski L, Lambert L, Foote J, Wallace MR, Kesterson RA, Dickson G, Popplewell L, Wallis D. Targeted exon skipping of NF1 exon 17 as a Therapeutic for Neurofibromatosis Type I. Mol Ther Nucleic Acids. 2022;[Epub] doi:10.1016/j.omtn.2022.03.011
    cell culture: human lines with NF1 e17 mutations, Morpholinos delivered using Endo-Porter DMSO

    Yu T, Cazares O, Tang AD, Kim HY, Wald T, Verma A, Liu Q, Barcellos-Hoff MH, Floor SN, Jung HS, Brooks AN, Klein OD. SRSF1 governs progenitor-specific alternative splicing to maintain adult epithelial tissue homeostasis and renewal. Dev Cell. 2022 Feb 22:S1534-5807(22)00036-3. doi: 10.1016/j.devcel.2022.01.011. Online ahead of print.
    cell culture: primary murine cells: dental epithelium, intestinal organoid crypt

    Catasus N, Rosas I, Bonache S, Negro A, Plana A, Salvador H, Serra E, Ignacio Blanco I, Castellanos E. Antisense oligonucleotides targeting exon 11 are able to partially rescue the Neurofibromatosis Type 2 phenotype in vitro. bioRxiv. 2022;[preprint] doi:10.1101/2022.02.11.479859
    cell culture: human primary neurofibromatosis cells

    Moriarty RA, Mili S, Stroka KM. RNA Localization in Confined Cells Depends on Cellular Mechanical Activity and Contributes to Confined Migration. iScience. 2022;[Epub ahead of print] doi:10.2139/ssrn.3878411

    Awad EK, Moore M, Liu H, Ciszewski L, Lambert L, Korf BR, Popplewell L, Kesterson RA, Wallis D. Restoration of Normal NF1 Function with Antisense Morpholino Treatment of Recurrent Pathogenic Patient-Specific Variant c.1466A>G; p.Y489C. J Pers Med 2021;11(12):1320. doi:10.3390/jpm11121320
    Endo-Porter (DMSO) delivery to human iPSC

    Price AM, Steinbock RT, Di C, Hayer KE, Li Y, Herrmann C, Parenti NA, Whelan JN, Weiss SR, Weitzman MD. Adenovirus prevents dsRNA formation by promoting efficient splicing of viral RNA. Nucl Acids Res. 2021;[Epub]:gkab896. doi:10.1093/nar/gkab896
    Endo-Porter PEG

    Todaro AM, Hackeng TM, Castoldi E. Antisense-Mediated Down-Regulation of Factor V-Short Splicing in a Liver Cell Line Model. Appl Sci. 2021;11(20):9621. doi:0.3390/app11209621
    Delivery to HepG2 cells with Endo-Porter

    Li Y, Garcia G Jr, Arumugaswami V, Guo F. Structure-based design of antisense oligonucleotides that inhibit SARS-CoV-2 replication. bioRxiv. 2021;[preprint] doi:10.1101/2021.08.23.457434
    cell culture: HEK293-hACE2

    Michaels WE, Pena-Rasgado C, Kotaria R, Bridges RJ, Hastings ML. Open reading frame correction using antisense oligonucleotides for the treatment of cystic fibrosis caused by CFTR-W1282X. bioRxiv. 2021;[preprint] doi:10.1101/2021.08.11.455834
    cell culture: CFTR-W1282X bronchial epithelial line, primary CFTR-W1282X epithelial cells

    Pradella D, Deflorian G, Pezzotta A, Di Matteo A, Belloni E, Campolungo D, Paradisi A, Bugatti M, Vermi W, Campioni M, Chiapparino A, Scietti L, Forneris F, Giampietro C, Volf N, Rehman M, Zacchigna S, Paronetto MP, Pistocchi A, Eichmann A, Mehlen P, Ghigna C. A ligand-insensitive UNC5B splicing isoform regulates angiogenesis by promoting apoptosis. Nat Comm. 2021;12(1):4872. doi:10.1038/s41467-021-24998-6
    cell culture: HUVEC/TERT2

    Wu B, Drains M, Lu P, Lu Q, Wang M. Neutral Surfactant Enhanced Exon-skipping of Morpholino Oligonucleotides in vitro and in mdx Mice. J Nanomed Nanotechnol. 2020;11(6):553. doi:10.35248/2157-7439.20.11.553
    Endo-Porter in C2C12E50 myoblasts and C2C12E23 differentiated cells expressing human dystrophin exon 50 sequence (hDyE50); mouse dystrophin exon 23 sequence (mDyE23) in the GFP coding sequence, respectively

    Fish L, Khoroshkin M, Navickas A, Garcia K, Culbertson B, Hänisch B, Zhang S, Nguyen CB, Soto LM, Dermit M, Mardakheh FK, Molina H, Alarcón C, Najafabadi HS, Goodarzi H. A prometastatic splicing program regulated by SNRPA1 interactions with structured RNA elements. Science. 2021;372(6543): doi:10.1126/science.abc7531
    Delivery into cultured cells by Endo-Porter, cells then tail-vein injected into mice for metastasis model

    Katow H, Katow T, Yoshida H, Kiyomoto M. Involvement of Huntingtin in Development and Ciliary Beating Regulation of Larvae of the Sea Urchin, Hemicentrotus pulcherrimus. Int J Mol Sci. 2021;22(10):5116. doi:10.3390/ijms22105116
    Delivery into sea urchin embryos using Endo-Porter PEG

    Biayna J, Mazuelas H, Gel B, Terribas E, Dumbovic G, Rosas I, Fernández-Rodriguez J, Blanco I, Castellanos E, Carrió M, Lazaro C, Serra E. Using antisense oligonucleotides for the physiological modulation of the alternative splicing of NF1 exon 23a during PC12 neuronal differentiation. Sci Rep. 2021;11(1):3661. doi:10.1038/s41598-021-83152-w

    Lanzarini F, Pereira FA, de Camargo J, Oliveira AM, Belaz KRA, Melendez-Perez JJ, Eberlin MN, Brum MCS, Mesquita FS, Sudano MJ. ELOVL5 Participates in Embryonic Lipid Determination of Cellular Membranes and Cytoplasmic Droplets. Int J Mol Sci. 2021;22(3):1311. doi:10.3390/ijms22031311
    Delivery by Endo-Porter to cattle embryos

    Ogawa T, Sato R, Naganuma T, Liu K, Sato S, Sakaue S, Osada M, Yoshimi K, Muramoto K. Diversified Biomineralization Roles of Pteria penguin Pearl Shell Lectins as Matrix Proteins. Int J Mol Sci. 2021;22(3),1081. doi:10.3390/ijms22031081
    Transfection into oyster larvae by Endo-Porter from artificial sea water

    Wang X, Liu L, Whisnant AW, Hennig T, Djakovic L, Haque N, Bach C, Sandri-Goldin RM, Erhard F, Friedel CC, Dölken L, Shi Y. Mechanism and consequences of herpes simplex virus 1-mediated regulation of host mRNA alternative polyadenylation. bioRxiv. 2020;[preprint] doi:10.1101/2020.11.26.399626
    Delivery into HEK293 cells

    Xu J, Wan Z, Tang M, Lin Z, Jiang S, Ji L, Gorshkov K, Mao Q, Xia S, Cen D, Zheng J, Liang X, Cai X. N6-methyladenosine-modified CircRNA-SORE sustains sorafenib resistance in hepatocellular carcinoma by regulating β-catenin signaling. Mol Cancer. 2020:19(1):163. doi:10.1186/s12943-020-01281-8
    Blocking an adenosine methylation site (N6) in a circular RNA with a Morpholino delivered into HepG2 cells by Endo-Porter

    Grocott T, Lozano-Velasco E, Mok GF, Münsterberg AE. The Pax6 master control gene initiates spontaneous retinal development via a self-organising Turing network. Development. 2020;[Epub ahead of print] doi:10.1242/dev.185827 Delivery to cultured Gallus gallus (chick) somite tissue with Endo-Porter PEG

    Bergsma AJ, in ’t Groen SLM, Catalano F, Yamanaka M, Takahashi S, Okumiya T, van der Ploeg AT, Pijnappel WWMP. A generic assay for the identification of splicing variants that induce nonsense-mediated decay in Pompe disease. Eur J Hum Genet. 2020;{Epub ahead of print]. doi:10.1038/s41431-020-00751-3
    Endo-Porter DMSO used for Morpholino delivery into primary human fibroblast cultures

    Li Y, Gao X, Wei C, Guo R, Xu H, Bai Z, Zhou J, Zhu J, Wang W, Wu Y, Li J, Zhang Z, Xie X. Modification of Mcl-1 alternative splicing induces apoptosis and suppresses tumor proliferation in gastric cancer. Aging. 2020;12. doi:10.18632/aging.103766
    gastric cancer lines MKN-28, SGC-7901, and MKN-45

    Brooks KE, Daughtry BL, Davis B, Yan MY, Fei SS, Carbone L, Chavez SL. Molecular Contribution to Embryonic Aneuploidy and Genotypic Complexity During Initial Cleavage Divisions of Mammalian Development. bioRxiv. 2020;[preprint] doi:10.1101/2020.07.24.220475 Madin-Darby Bovine Kidney (MDBK) epithelial line, Morpholinos delivered using Endo-Porter DMSO

    Lobo S, Pereira C, Oliveira C, Almeida GM. Skipping Exon-v6 from CD44v6-Containing Isoforms Influences Chemotherapy Response and Self-Renewal Capacity of Gastric Cancer Cells. Cancers. 2020;12(9):2072-6694 doi:10.3390/cancers12092378
    cell culture: human gastric cancer, MKN45 & GP202

    Korecka JA, Thomas R, Hinrich AJ, Moskites AM, Macbain ZK, Hallett PJ, Isacson O, Hastings ML. Splice-Switching Antisense Oligonucleotides Reduce LRRK2 Kinase Activity in Human LRRK2 Transgenic Mice. Mol Ther Nucleic Acids. 2020 Jun 27;21:623-635. doi: 10.1016/j.omtn.2020.06.027. Online ahead of print.
    cell culture: patient-derived fibroblasts

    Mirando AC, Lima e Silva R, Chu Z, Campochiaro PA, Pandey NB, Popel AS. Suppression of Ocular Vascular Inflammation through Peptide-Mediated Activation of Angiopoietin-Tie2 Signaling. Int J Molec Sci. 2020;21(14):1422-0067 doi:10.3390/ijms21145142
    Cell culture: HUVEC

    Li L, Hobson L, Perry L, Clark B, Heavey S, Haider A, Sridhar A, Shaw G, Kelly J, Freeman A, Wilson I, Whitaker H, Nurmemmedov E, Oltean S, Porazinski S, Ladomery M. Targeting the ERG oncogene with splice-switching oligonucleotides as a novel therapeutic strategy in prostate cancer. Br J Cancer. 2020;[Epub ahead of print]doi:10.1038/s41416-020-0951-2. Online ahead of print.
    Cell culture MG63, VCaP; xenograft mice; primary human tumor explant. Vivo-Morpholinos used with Endo-Porter in VCaP cells.

    Rocher C, Vernale A, Fierro-Constain L, Sejourne N, Chenesseau S, Marschal C, Le Golf E, Dutilleul M, Matthews C, Marschal F, Brouilly N, Massey-Harroche D, Ereskovsky A, Le Bivic A, Borchiellini C, Renard E. The buds of Oscarella lobularis (Porifera): a new convenient model for sponge cell and developmental biology. bioRxiv. 2020;[preprint] doi:10.1101/2020.06.23.167296

    Hara Y, Mizobe Y, Inoue YU, Hashimoto Y, Motohashi N, Masaki Y, Seio K, Takeda S, Nagata T, Wood MJA, Inoue T, Aoki Y. Novel EGFP reporter cell and mouse models for sensitive imaging and quantification of exon skipping. Sci Rep. 2020;10(1):10110. doi: 10.1038/s41598-020-67077-4

    Escobar-Hoyos LF, Penson A, Kannan R, Cho H, Pan C-H, Singh RK, Apken LK, Hobbs GA, Luo R, Lecomte N, Babu S, Pan FC, Alonso-Curbelo D, Morris JP, Askan G, Grbovic-Huezo O, Ogrodowski P, Bermeo J, Saglimbeni J, Cruz CD, Ho Y-J, Lawrence SA, Melchor JP, Goda Ga, Bai K, Pastore A, Hogg SJ, Raghavan S, Bailey P, Chang DK, Biankin A, Shroyer KR, Wolpin BM, Aguirre AJ, Ventura A, Taylor B, Der CJ, Dominguez D, Kümmel D, Oeckinghaus A, Lowe SW, Bradley RK, Abdel-Wahab O, Leach SD. Altered RNA Splicing by Mutant p53 Activates Oncogenic RAS Signaling in Pancreatic Cancer. Cancer Cell. 2020;[Epub ahead of print] doi:10.1016/j.ccell.2020.05.010

    Michaels WE, Bridges RJ, Hastings ML. Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells. Nucl Acids Res. 2020;[Epub ahead of print] doi:10.1093/nar/gkaa490

    Takizawa H, Sato M, Aoki Y. Exon Skipping in Directly Reprogrammed Myotubes Obtained from Human Urine-Derived Cells. J Vis Exp. 2020;(159):e60840. doi:10.3791/60840
    Delivery into myotubes from human urine-derived cells

    Michaels WE, Bridges RJ, Hastings ML. Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells. bioRxiv. 2020;[preprint] doi:10.1101/2020.05.12.089417
    cell culture: lymphoblast cell lines, hBE cells

    Tanaka I, Chakraborty A, Saulnier O, Benoit-Pilven C, Vacher S, Labiod D, Lam EWF, Bièche I, Delattre O, Pouzoulet F, Auboeuf D, Vagner S, Dutertre M. ZRANB2 and SYF2-mediated splicing programs converging on ECT2 are involved in breast cancer cell resistance to doxorubicin. Nucleic Acids Res. 2020 Jan 16. pii: gkz1213. doi: 10.1093/nar/gkz1213. [Epub ahead of print]
    cell culture: MCF7-DoxoR Endo-Porter PEG

    Fiszbein A, Krick KS, Begg BE, Burge CB. Exon-Mediated Activation of Transcription Starts. Cell. 2019;[epub ahead of print] doi:10.1016/j.cell.2019.11.002
    cell culture: mouse fibroblasts

    Willson JA, Bork BS, Muir CA, Damjanovski S. Modulation of RECK levels in Xenopus A6 cells: effects on MT1-MMP, MMP-2 and pERK levels. J Biol Res (Thessaloniki). 2019;26:16. doi:10.1186/s40709-019-0108-8
    cell culture: Xenopus A6 cells

    Haririan M, Kaviani S, Soleimani M, Noruzinia M, Abroun S, Rasi Ghaemi S, Delalat B. [Evaluation of Morpholino antisense oligos' role on BCR-ABL gene silencing in the K562 cell line]. Yakhteh Medical Journal. 2010;12(2):241-248.

    de Morree A, Klein JDD, Gan Q, Farup J, Urtasun A, Kanugovi A, Bilen B, van Velthoven CTJ, Quarta M, Rando TA. Alternative polyadenylation of Pax3 controls muscle stem cell fate and muscle function. Science. 2019 Nov 8;366(6466):734-738. doi: 10.1126/science.aax1694.
    cell culture: mouse muscle stem cells (MuSC)

    Pritchard HAT, Griffin CS, Yamasaki E, Thakore P, Lane C, Greenstein AS, Earley S. Nanoscale coupling of junctophilin-2 and ryanodine receptors regulates vascular smooth muscle cell contractility. PNAS USA. 2019;[Epub ahead of print] doi:10.1073/pnas.1911304116
    Delivery into dissected cerebral arteries (vascular smooth muscle)

    Sato M, Shiba N, Miyazaki D, Shiba Y, Echigoya Y, Yokota T, Takizawa H, Aoki Y, Takeda S, Nakamura A. Amelioration of intracellular Ca2+ regulation by exon-45 skipping in Duchenne muscular dystrophy-induced pluripotent stem cell-derived cardiomyocytes. Biochem Biophys Res Commun. 2019 Oct 1. pii: S0006-291X(19)31825-X. doi: 10.1016/j.bbrc.2019.09.095. [Epub ahead of print]
    Delivery to iPSC-derived cardiomyocytes

    Takada-Takatori Y, Nakagawa S, Kimata R, Nao Y, Mizukawa Y, Urushidani T, Izumi Y, Akaike A, Tsuchida K, Kume T. Donepezil modulates amyloid precursor protein endocytosis and reduction by up-regulation of SNX33 expression in primary cortical neurons. Sci Rep. 2019;9:11922. doi:10.1038/s41598-019-47462-4

    Hoss F, Mueller JL, Rojas Ringeling F, Rodriguez-Alcazar JF, Brinkschulte R, Seifert G, Stahl R, Broderick L, Putnam CD, Kolodner RD, Canzar S, Geyer M, Hoffman HM, Latz E. Alternative splicing regulates stochastic NLRP3 activity. Nat Commun. 2019 Jul 19;10(1):3238. doi: 10.1038/s41467-019-11076-1.
    human monocyte-derived macrophages (hMDMs)

    Molinari E, Ramsbottom SA, Srivastava S, Booth P, Alkanderi S, McLafferty SM, Devlin LA, White K, Gunay-Aygun M, Miles CG, Sayer JA. Targeted exon skipping rescues ciliary protein composition defects in Joubert syndrome patient fibroblasts. Sci Rep. 2019 Jul 25;9(1):10828. doi: 10.1038/s41598-019-47243-z.
    primary human fibroblasts

    Lee JJA, Saito T, Duddy W, Takeda S, Yokota T. Direct Reprogramming of Human DMD Fibroblasts into Myotubes for In Vitro Evaluation of Antisense-Mediated Exon Skipping and Exons 45-55 Skipping Accompanied by Rescue of Dystrophin Expression. Methods Mol Biol. 2018;1828:141-150. doi: 10.1007/978-1-4939-8651-4_8.

    Wang M, Wu B, Shah SN, Lu P, Lu Q. Aminoglycoside Enhances the Delivery of Antisense Morpholino Oligonucleotides in vitro and in mdx Mice. Molec Ther Nucl Acids. 2019;[Epub ahead of print] doi:10.1016/j.omtn.2019.04.023

    Bolduc V, Foley AR, Solomon-Degefa H, Sarathy A, Donkervoort S, Hu Y, Chen GS, Sizov K, Nalls M, Zhou H, Aguti S, Cummings BB, Lek M, Tukiainen T, Marshall JL, Regev O, Marek-Yagel D, Sarkozy A, Butterfield RJ, Jou C, Jimenez-Mallebrera C, Li Y, Gartioux C, Mamchaoui K, Allamand V, Gualandi F, Ferlini A, Hanssen E; COL6A1 Intron 11 Study Group, Wilton SD, Lamandé SR, MacArthur DG, Wagener R, Muntoni F, Bönnemann CG. A recurrent COL6A1 pseudoexon insertion causes muscular dystrophy and is effectively targeted by splice-correction therapies. JCI Insight. 2019 Mar 21;4(6). pii: 124403. doi: 10.1172/jci.insight.124403. eCollection 2019 Mar 21.
    Delivery to human dermal fibroblasts & HEK293T

    Neumann A, Schindler M, Olofsson D, Wilhelmi I, Schürmann A, Heyd F. Genome-wide identification of alternative splicing events that regulate protein transport across the secretory pathway. J Cell Sci. 2019 Mar 19. pii: jcs.230201. doi: 10.1242/jcs.230201. [Epub ahead of print]
    Delivery to HEK293T cultures

    Angiolini F, Belloni E, Giordano M, Campioni M, Forneris F, Paola PM, Lupia M, Brandas C, Pradella D, Di Matteo A, Giampietro C, Jodice G, Luise C, Bertalot G, Freddi S, Malinverno M, Irimia M, Moulton JD, Summerton J, Chiapparino A, Ghilardi C, Giavazzi R, Nyqvist D, Gabellini D, Dejana E, Cavallaro U, Ghigna C. A novel L1CAM isoform with angiogenic activity generated by NOVA2-mediated alternative splicing. Elife. 2019 Mar 4;8. pii: e44305. doi: 10.7554/eLife.44305.
    cell culture: mouse lu2EC, Morpholinos delivered with Endo-Porter PEG

    Fiszbein A, Krick KS, Burge CB. Exon-mediated activation of transcription starts. bioRxiv. 2019;[preprint] doi:10.1101/565184
    cell culture: mouse fibroblasts

    Korecka JA, Talbot S, Osborn TM, de Leeuw SM, Levy SA, Ferrari EJ, Moskites A, Atkinson E, Jodelka FM, Hinrich AJ, Hastings ML, Woolf CJ, Hallett PJ, Isacson O. Neurite Collapse and Altered ER Ca2+ Control in Human Parkinson Disease Patient iPSC-Derived Neurons with LRRK2 G2019S Mutation. Stem Cell Reports. 2018 Dec 14. pii: S2213-6711(18)30490-9. doi: 10.1016/j.stemcr.2018.11.021. [Epub ahead of print]
    cell culture: human Parkinson's Disease iPSC-derived neurons

    Blazquez L, Emmett W, Faraway R, Pineda JMB, Bajew S, Gohr A, Haberman N, Sibley CR, Bradley RK, Irimia M, Ule J. Exon Junction Complex Shapes the Transcriptome by Repressing Recursive Splicing. Mol Cell. 2018 Nov 1;72(3):496-509.e9. doi: 10.1016/j.molcel.2018.09.033.
    HeLa Flp-In T-REx KPNA1, cultured mouse neural precursos cells

    Ramsbottom SA, Molinari E, Srivastava S, Silberman F, Henry C, Alkanderi S, Devlin LA, White K, Steel DH, Saunier S, Miles CG, Sayer JA. Targeted exon skipping of a CEP290 mutation rescues Joubert syndrome phenotypes in vitro and in a murine model. Proc Natl Acad Sci U S A. 2018 Nov 16. pii: 201809432. doi: 10.1073/pnas.1809432115. [Epub ahead of print]
    Delivery into human urine-derived renal epithelial kidney cells (hURECs)

    Watanabe N, Nagata T, Satou Y, Masuda S, Saito T, Kitagawa H, Komaki H, Takagaki K, Takeda S. NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids. 2018 Sep 27;13:442-449. doi: 10.1016/j.omtn.2018.09.017. [Epub ahead of print]

    Lee JJA, Maruyama R, Duddy W, Sakurai H, Yokota T. Identification of novel antisense-mediated exon skipping targets in DYSF for therapeutic treatment of dysferlinopathy. Molec Ther Nucleic Acids. 2018;[Epub ahead of print] doi:10.1016/j.omtn.2018.10.004

    Belanger AM, Przybylska M, Gefteas E, Furgerson M, Geller S, Kloss A, Cheng SH, Zhu Y, Yew NS. Inhibiting neutral amino acid transport for the treatment of phenylketonuria. JCI Insight. 2018 Jul 26;3(14). pii: 121762. doi: 10.1172/jci.insight.121762. [Epub ahead of print]

    Shimo T, Tachibana K, Obika S. Construction of a tri-chromatic reporter cell line for the rapid and simple screening of splice-switching oligonucleotides targeting DMD exon 51 using high content screening. PLoS One. 2018 May 16;13(5):e0197373. doi: 10.1371/journal.pone.0197373. eCollection 2018.
    cell culture: RD

    Wang M, Wu B, Shah SN, Lu P, Lu Q. Saponins as Natural Adjuvant for Antisense Morpholino Oligonucleotides Delivery in vitro and in mdx Mice. Molec Ther Nucl Acids. 2018;[Epub ahead of print] doi:10.1016/j.omtn.2018.02.004
    cell culture: C2C12E50, C2C12E23

    Lenis YY, Johnson GA, Wang X, Tang WW, Dunlap KA, Satterfield MC, Wu G, Hansen TR, Bazer FW. Functional roles of ornithine decarboxylase and arginine decarboxylase during the peri-implantation period of pregnancy in sheep. J Anim Sci Biotechnol. 2018 Jan 24;9:10. doi: 10.1186/s40104-017-0225-x. eCollection 2018.
    "Briefly, a small incision was made in the oviduct just above the tubouterine junction and a small catheter was passed through the incision and into the uterine lumen. The catheter was attached to a 1-mL syringe containing the morpholino and the morpholino was delivered from the syringe into the uterine lumen. ... The MAO combination (100 nmol MAO-ODC1 plus 100 nmol MAO-ADC) was prepared with Gene Tools Endo-Porter delivery reagent (200 μL) and diluted to 1.2 mL final volume with OPTI-MEM (Invitrogen; Grand Island, NY, U.S.A.). All MAOs were injected once into the lumen of the uterine horn (cranial portion) ipsilateral to the ovary with a corpus luteum (CL)."

    Wang M, Wu B, Tucker JD, Shah SN, Lu P, Bollinger LE, Lu Q. Tween 85-Modified Low Molecular Weight PEI Enhances Exon-Skipping of Antisense Morpholino Oligomer In Vitro and in mdx Mice. Mol Ther Nucleic Acids. 2017 Dec 15;9:120-131. doi: 10.1016/j.omtn.2017.09.006. Epub 2017 Sep 20.
    Delivery by Tween 85-Modified PEI, used Endo-Porter as a delivery control

    Yaparla A, Popovic M, Grayfer L. Differentiation-dependent antiviral capacities of amphibian (Xenopus laevis) macrophages. J Biol Chem. 2017 Dec 19. pii: jbc.M117.794065. doi: 10.1074/jbc.M117.794065. [Epub ahead of print].
    Delivery to X. laevis kidney (A6) cell line using Endo-Porter PEG

    Morrison JA, McLennan R, Wolfe LA, Gogol MM, Meier S, McKinney MC, Teddy JM, Holmes L, Semerad CL, Box AC, Li H, Hall KE, Perera AG, Kulesa PM. Single-cell transcriptome analysis of avian neural crest migration reveals signatures of invasion and molecular transitions. Elife. 2017 Dec 4;6. pii: e28415. doi: 10.7554/eLife.28415.
    Delivery into LMH cell line from Gallus gallus (chick)

    Adamo P, Porazinski S, Rajatileka S, Jumbe S, Hagen R, Cheung MK, Wilson I, Ladomery MR. The oncogenic transcription factor ERG represses the transcription of the tumour suppressor gene PTEN in prostate cancer cells. Oncol Lett. 2017 Nov;14(5):5605-5610. doi: 10.3892/ol.2017.6841. Epub 2017 Aug 28.
    Vivo-Morpholinos combined with Endo-Porter in VCaP (ATCC CRL-2876) cell culture

    Fagg WS, Liu N, Fair JH, Shiue L, Katzmann S, Donohue J-P, Ares M. Autogenous Cross-Regulation Of Quaking mRNA Processing And Translation Balances Quaking Functions In Splicing And Translation. bioRxiv. 2017.
    Endo-Porter used in the presence of 1% pen/strep.

    Egesipe AL, Blondel S, Cicero AL, Jaskowiak AL, Navarro C, Sandre-Giovannoli A, Levy N, Peschanski M, Nissan X. Metformin decreases progerin expression and alleviates pathological defects of Hutchinson-Gilford progeria syndrome cells. NPJ Aging Mech Dis. 2016 Nov 10;2:16026. doi: 10.1038/npjamd.2016.26. eCollection 2016.
    cell culture: Hutchinson-Gilford progeria syndrome mesenchymal stem cells

    Harhouri K, Navarro C, Depetris D, Mattei MG, Nissan X, Cau P, De Sandre-Giovannoli A, Lévy N. MG132-induced progerin clearance is mediated by autophagy activation and splicing regulation. EMBO Mol Med. 2017 Jul 3. pii: e201607315. doi: 10.15252/emmm.201607315. [Epub ahead of print]

    van der Wal E, Bergsma AJ, Pijnenburg JM, van der Ploeg AT, Pijnappel WWMP. Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease. Mol Ther Nucleic Acids. 2017 Jun 16;7:90-100. doi: 10.1016/j.omtn.2017.03.001. Epub 2017 Mar 14.
    cell culture: human primary fibroblasts

    Witola WH, Zhang X, Kim CY. Targeted gene knockdown validates the essential role of lactate dehydrogenase in Cryptosporidium parvum. Int J Parasitol. 2017 Jun 9. pii: S0020-7519(17)30166-2. doi: 10.1016/j.ijpara.2017.05.002. [Epub ahead of print]

    Fimiani C, Goina E, Su Q, Gao G, Mallamaci A. RNA activation of haploinsufficient Foxg1 gene in murine neocortex. Sci Rep. 2016 Dec 20;6:39311. doi: 10.1038/srep39311.

    Egesipe A-L, Blondel S, Cicero AL, Jaskowiak A-L, Navarro C, De Sandre-Giovannoli A, Levy N, Peschanski M, Nissan X. Metformin decreases progerin expression and alleviates pathological defects of Hutchinson-Gilford progeria syndrome cells. npj Aging and Mechanisms of Disease. 2016;2:16026. doi:10.1038/npjamd.2016.26.
    Delivery into mesenchymal stem cells derived from HGPS iPS cells

    Yuan Y, Wang Y, Liu Q, Zhu F, Hong Y. Singapore grouper iridovirus protein VP088 is essential for viral infectivity. Sci Rep. 2016 Aug 8;6:31170. doi: 10.1038/srep31170.
    cell culture: Oryzias latipes (Medaka) haploid ES cell line HX1

    Wilhelmi I, Kanski R, Neumann A, Herdt O, Hoff F, Jacob R, Preußner M, Heyd F. Sec16 alternative splicing dynamically controls COPII transport efficiency . Nat Commun. 2016 Aug 5;7:12347. doi: 10.1038/ncomms12347.
    cell culture: Hek293 and HeLa and human peripheral blood mononuclear cells (PBMCs) enriched for T cells

    Uzureau S, Coquerelle C, Vermeiren C, Uzureau P, Van Acker A, Pilotte L, Monteyne D, Acolty V, Vanhollebeke B, Van den Eynde B, Pérez-Morga D, Moser M, Pays E. Apolipoproteins L control cell death triggered by TLR3/TRIF signaling in dendritic cells. Eur J Immunol. 2016 Aug;46(8):1854-66. doi: 10.1002/eji.201546252. Epub 2016 Jun 8.
    cell culture: CD8α+ MutuDCs (dendritic cells)

    Wang M, Wu B, Tucker JD, Bollinger LE, Lu P, Lu Q. Poly(ester amine) Composed of Polyethylenimine and Pluronic Enhance Delivery of Antisense Oligonucleotides In Vitro and in Dystrophic mdx Mice. Mol Ther Nucleic Acids. 2016 Aug 2;5(8):e341. doi: 10.1038/mtna.2016.51.
    cell culture: C2C12E50

    Fraszczak J, Helness A, Chen R, Vadnais C, Robert F, Khandanpour C, Möröy T. Threshold Levels of Gfi1 Maintain E2A Activity for B Cell Commitment via Repression of Id1. PLoS One. 2016;11(7):e0160344. doi:10.1371/journal.pone.0160344
    Endo-Porter delivery into cultured LSK cells (B cell progenitors)

    Osman EY, Washington CW 3rd, Kaifer KA, Mazzasette C, Patitucci TN, Florea KM, Simon ME, Ko CP, Ebert AD, Lorson CL. Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy. Mol Ther. 2016 Jul 12. doi: 10.1038/mt.2016.145. [Epub ahead of print]
    Endo-Porter delivery into iPS-derived SMA motor neurons

    Harhouri K, Navarro C, Baquerre C, Da Silva N, Bartoli C, Casey F, Mawuse GK, Doubaj Y, Lévy N, De Sandre-Giovannoli A. Antisense-Based Progerin Downregulation in HGPS-Like Patients’ Cells. Cells, 2016;5(3):31. doi:10.3390/cells5030031

    Sato Y, Sato S, Kikuchi T, Nonaka A, Kumagaia Y, Sasaki A, Kobayashi M. Knockdown of gene expression by antisense morpholino oligos in preimplantation mouse embryos cultured in vitro. Anal Biochem. 2016;[Epub ahead of print] doi:10.1016/j.ab.2016.06.032

    Parfitt DA, Lane A, Ramsden CM, Carr A-JF, Munro PM, Jovanovic K, Schwarz N, Kanuga N, Muthiah MN, Hull S, Gallo J-M, da Cruz L, Moore AT, Hardcastle AJ, Coffey PJ, Cheetham ME. Identification and Correction of Mechanisms Underlying Inherited Blindness in Human iPSC-Derived Optic Cups. Cell Stem Cell. 2016;[Epub ahead of print] doi:10.1016/j.stem.2016.03.021
    human fibroblast-derived iPSC

    Vad-Nielsen J, Jakobsen KR, Daugaard TF, Thomsen R, Brügmann A, Sørensen BS, Nielsen AL. Regulatory dissection of the CBX5 and hnRNPA1 bi-directional promoter in human breast cancer cells reveals novel transcript variants differentially associated with HP1α down-regulation in metastatic cells. BMC Cancer. 2016;16:32. doi:10.1186/s12885-016-2059-x
    cell culture: MDA-MB-231 cells

    Brooks K, Burns G, Spencer TE. Biological Roles of Hydroxysteroid (11-Beta) Dehydrogenase 1 (HSD11B1), HSD11B2, and Glucocorticoid Receptor (NR3C1) in Sheep Conceptus Elongation. Biol Reprod. 2015 Jun 17. pii: biolreprod.115.130757. [Epub ahead of print]
    Delivery in uterus to endometrium of sheep

    Gao M, Yan H, Yin RH, Wang Q, Zhan YQ, Yu M, Ge CH, Li CY, Wang XH, Ge ZQ, Yang XM. Hepassocin is required for hepatic outgrowth during zebrafish hepatogenesis. Biochem Biophys Res Commun. 2015 Jun 3. pii: S0006-291X(15)30045-0. doi: 10.1016/j.bbrc.2015.05.121. [Epub ahead of print]
    cell culture: delivery to HEK293T cells

    Curtis AM, Fagundes CT, Yang G, Palsson-McDermott EM, Wochal P, McGettrick AF, Foley NH, Early JO, Chen L, Zhang H, Xue C, Geiger SS, Hokamp K, Reilly MP, Coogan AN, Vigorito E, FitzGerald GA, O'Neill LA. Circadian control of innate immunity in macrophages by miR-155 targeting Bmal1. Proc Natl Acad Sci U S A. 2015 May 20. pii: 201501327. [Epub ahead of print]
    cell culture: mouse bone marrow derived macrophages BMDM (primary)

    Mino T, Murakawa Y, Fukao A, Vandenbon A, Wessels HH, Ori D, Uehata T, Tartey S, Akira S, Suzuki Y, Vinuesa CG, Ohler U, Standley DM, Landthaler M, Fujiwara T, Takeuchi O. Regnase-1 and Roquin Regulate a Common Element in Inflammatory mRNAs by Spatiotemporally Distinct Mechanisms. Cell. 2015 May 21;161(5):1058-73. doi: 10.1016/j.cell.2015.04.029.

    Wang K, Liu CY, Zhou LY, Wang JX, Wang M, Zhao B, Zhao WK, Xu SJ, Fan LH, Zhang XJ, Feng C, Wang CQ, Zhao YF, Li PF. APF lncRNA regulates autophagy and myocardial infarction by targeting miR-188-3p. Nat Commun. 2015 Apr 10;6:6779. doi: 10.1038/ncomms7779.
    cell culture: mouse primary cardiomyocytes

    Echigoya Y, Mouly V, Garcia L, Yokota T, Duddy W. In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in duchenne muscular dystrophy. PLoS One. 2015 Mar 27;10(3):e0120058. doi: 10.1371/journal.pone.0120058. eCollection 2015.
    cell culture: human myotubes

    Becerra S, Montes M, Hernández-Munain C, Suñé C. Prp40 pre-mRNA processing factor 40 homolog B (PRPF40B) associates with SF1 and U2AF65 and modulates alternative pre-mRNA splicing in vivo. RNA. 2015 Mar;21(3):438-57. doi: 10.1261/rna.047258.114. Epub 2015 Jan 20.
    cell culture: HEK293T cells

    Yoshida M, Kitaoka S, Egawa N, Yamane M, Ikeda R, Tsukita K, Amano N, Watanabe A, Morimoto M, Takahashi J, Hosoi H, Nakahata T, Inoue H, Saito MK. Modeling the Early Phenotype at the Neuromuscular Junction of Spinal Muscular Atrophy Using Patient-Derived iPSCs. Stem Cell Rep. 2015;[Epub ahead of print]. 10.1016/j.stemcr.2015.02.010
    cell culture: human iPSC co-culture with C2C12, delivery by Endo-Porter

    Valiunas V, Wang HZ, Li L, Gordon C, Valiuniene L, Cohen IS, Brink PR. A comparison of two cellular delivery mechanisms for small interfering RNA. Physiol Rep. 2015 Feb 3;3(2). pii: e12286. doi: 10.14814/phy2.12286. Print 2015 Feb 1.

    Brooks KE, Burns GW, Spencer TE. Peroxisome Proliferator Activator Receptor Gamma (PPARG) Regulates Conceptus Elongation in Sheep. Biol Reprod. 2014 Dec 17. pii: biolreprod.114.123877. [Epub ahead of print]
    Endo-Porter (EP) in culture, intrauterine infusion with EP

    Brooks K, Spencer TE. Biological Roles of Interferon Tau (IFNT) and Type I IFN Receptors in Elongation of the Ovine Conceptus. Biol Reprod. 2014 Dec 10. pii: biolreprod.114.124156. [Epub ahead of print]
    Endo-Porter (EP) in culture, intrauterine injection with EP, intrauterine infusion with EP

    Naganuma T, Hoshino W, Shikanai Y, Sato R, Liu K, Sato S, Muramoto K, Osada M, Yoshima K, Ogawa T. Novel Matrix Proteins of Pteria penguin Pearl Oyster Shell Nacre Homologous to the Jacalin-Related β-Prism Fold Lectins. PLoS ONE. 2014;9(11):e112326. doi:10.1371/journal.pone.0112326
    Endo-Porter delivery of Morpholino from artificial sea water

    Gallego-Villar L, Viecelli HM, Pérez B, Harding CO, Ugarte M, Thöny B, Desviat LR. A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liver. Mol Ther Nucleic Acids. 2014 Sep 16;3:e193. doi: 10.1038/mtna.2014.44.

    Wang X, Frank JW, Xu J, Dunlap KA, Satterfield MC, Burghardt RC, Romero JJ, Hansen TR, Wu G, Bazer FW. Functional Role of Arginine During the Peri-Implantation Period of Pregnancy. II. Consequences of Loss of Function of Nitric Oxide Synthase NOS3 mRNA in Ovine Conceptus Trophectoderm. Biol Reprod. 2014 Jul 24. pii: biolreprod.114.121202. [Epub ahead of print]
    Endo-Porter in uterine horns of sheep (Ovis aries)

    Konantz J, Antos CL. Reverse Genetic Morpholino Approach Using Cardiac Ventricular Injection to Transfect Multiple Difficult-to-target Tissues in the Zebrafish Larva. J Vis Exp. 2014;88:e51595 doi:10.3791/51595
    Injections to zebrafish larvae

    Kalogeris TJ, Baines C, Korthuis RJ. Adenosine Prevents TNFα-Induced Decrease in Endothelial Mitochondrial Mass via Activation of eNOS-PGC-1α Regulatory Axis. PLoS ONE. 2014;9(6):e98459. doi:10.1371/journal.pone.0098459 Delivery to cultured human dermal microvascular endothelial cells (HMEC-1)

    Joshi HP, Subramanian IV, Schnettler EK, Ghosh G, Rupaimoole R, Evans C, Saluja M, Jing Y, Cristina I, Roy S, Zeng Y, Shah VH, Sood AK, Ramakrishnan S. Dynamin 2 along with microRNA-199a reciprocally regulate hypoxia-inducible factors and ovarian cancer metastasis. PNAS. 2014;[Epub ahead of print] doi:10.1073/pnas.1317242111

    Wang X, Ying W, Dunlap KA, Lin G, Satterfield MC, Burghardt RC, Wu G, Bazer FW. Arginine Decarboxylase and Agmatinase: An Alternative Pathway for De Novo Biosynthesis of Polyamines for Development of Mammalian Conceptuses. Biol Reprod. 2014 Mar 19. [Epub ahead of print]
    Endo-Porter in uterine horns of sheep (Ovis aries)

    Wang X, Frank JW, Little DR, Dunlap KA, Satterfield MC, Burghardt RC, Hansen TR, Wu G, Bazer FW. Functional role of arginine during the peri-implantation period of pregnancy. I. Consequences of loss of function of arginine transporter SLC7A1 mRNA in ovine conceptus trophectoderm. FASEB J. 2014;[Epub ahead of print] doi:10.1096/fj.13-248757
    Endo-Porter in sheep conceptus trophectoderm (Ovis aries)

    Wang K, Liu F, Zhou LY, Long B, Yuan SM, Wang Y, Liu CY, Sun T, Zhang XJ, Li PF. A Long Noncoding RNA, CHRF Regulates Cardiac Hypertrophy by Targeting miR-489. Circ Res. 2014 Feb 20. [Epub ahead of print]
    miRNA target-protecting Morpholino delivered to cultured cardiomyocytes with Endo-Porter

    Wang P, Calise J, Powell K, Divald A, Powell SR. Upregulation of proteasome activity rescues cardiomyocytes following pulse treatment with a proteasome inhibitor. Am J Cardiovasc Dis. 2014 Jan 15;4(1):6-13. eCollection 2014.
    Morpholinos delivered to rat neonatal cardiomyocytes in cell culture using Endo-Porter

    Krautbauer S, Eisinger K, Neumeier M, Hader Y, Buettner R, Schmid PM, Aslanidis C, Buechler C. Free Fatty Acids, Lipopolysaccharide and IL-1α Induce Adipocyte Manganese Superoxide Dismutase Which Is Increased in Visceral Adipose Tissues of Obese Rodents. PLoS One. 2014 Jan 24;9(1):e86866. doi: 10.1371/journal.pone.0086866. eCollection 2014.
    Endo-Porter used to deliver siRNA into predifferentiated 3T3-L1 cells

    Orrego PR, Olivares H, Cordero EM, Bressan A, Cortez M, Sagua H, Neira I, González J, da Silveira JF, Yoshida N, Araya JE. A Cytoplasmic New Catalytic Subunit of Calcineurin in Trypanosoma cruzi and Its Molecular and Functional Characterization. PLoS Negl Trop Dis. 2014;8(1):e2676. doi:10.1371/journal.pntd.0002676
    Morpholino delivery into Trypanosomes by Endo-porter

    Agranat-Tamir L, Shomron N, Sperling J, Sperling R. Interplay between pre-mRNA splicing and microRNA biogenesis within the supraspliceosome. Nucl Acids Res. 2014;[Epub ahead of print] doi:10.1093/nar/gkt1413
    Delivery into HeLa cultures

    Wang K, Liu F, Zhou LY, Ding SL, Long B, Liu CY, Sun T, Fan YY, Sun L, Li PF. miR-874 regulates myocardial necrosis by targeting caspase-8. Cell Death Dis. 2013 Jul 4;4:e709. doi: 10.1038/cddis.2013.233.

    Koebis M, Kiyatake T, Yamaura H, Nagano K, Higashihara M, Sonoo M, Hayashi Y, Negishi Y, Endo-Takahashi Y, Yanagihara D, Matsuda R, Takahashi MP, Nishino I, Ishiura S. Ultrasound-enhanced delivery of Morpholino with Bubble liposomes ameliorates the myotonia of myotonic dystrophy model mice. Sci Rep. 2013 Jul 22;3:2242. doi: 10.1038/srep02242.
    Knockdown in COS-7 cell culture

    Aouadi M, Tencerova M, Vangala P, Yawe JC, Nicoloro SM, Amano SU, Cohen JL, Czech MP. Gene silencing in adipose tissue macrophages regulates whole-body metabolism in obese mice. Proc Natl Acad Sci U S A. 2013 May 14;110(20):8278-83. doi: 10.1073/pnas.1300492110. Epub 2013 Apr 29.
    Packaged with siRNA into glucan shells, delivered into mice

    El-Gohary Y, Tulachan S, Guo P, Welsh C, Wiersch J, Prasadan K, Paredes J, Shiota C, Xiao X, Wada Y, Diaz M, Gittes G. Smad signaling pathways regulate pancreatic endocrine development. Dev Biol. 2013 Jun 15;378(2):83-93. doi: 10.1016/j.ydbio.2013.04.003. Epub 2013 Apr 17.
    Knockdown in hanging-drop cultures of mouse pancreata

    Kaur S, Soto-Pantoja DR, Stein EV, Liu C, Elkahloun AG, Pendrak ML, Nicolae A, Singh SP, Nie Z, Levens D, Isenberg JS, Roberts DD. Thrombospondin-1 Signaling through CD47 Inhibits Self-renewal by Regulating c-Myc and Other Stem Cell Transcription Factors. Sci Rep. 2013 Apr 17;3:1673. doi: 10.1038/srep01673.
    Delivering Morpholinos into primary mouse T-cells.

    Zammarchi F, Boutsalis G, Cartegni L. 5′ UTR Control of Native ERG and of Tmprss2:ERG Variants Activity in Prostate Cancer. PLoS ONE. 2013;8(3):e49721. doi:10.1371/journal.pone.0049721

    Patel M, Cai Q, Ding D, Salvi R, Hu Z, Hu BH. The miR-183/Taok1 Target Pair Is Implicated in Cochlear Responses to Acoustic Trauma. PLoS ONE. 2013;8(3):e58471. doi:10.1371/journal.pone.0058471
    Knockdown in rat cochlear explant cultures

    Mizrahi RA, Schirle NT, Beal PA. Potent and Selective Inhibition of A-to-I RNA Editing with 2’-O-Methyl/Locked Nucleic Acid-containing Antisense Oligoribonucleotides. ACS Chem Biol. 2013;[Epub ahead of print] doi:10.1021/cb300692k
    Morpholinos delivered into HeLa culture

    Pérez B, Gutiérrez-Solana LG, Verdú A, Merinero B, Yuste-Checa P, Ruiz-Sala P, Calvo R, Jalan A, Marín LL, Campos O, Ruiz MA, Miguel MS, Vázquez M, Castro M, Ferrer I, Navarrete R, Desviat LR, Lapunzina P, Ugarte M, Pérez-Cerdá C. Clinical, biochemical, and molecular studies in pyridoxine-dependent epilepsy. Antisense therapy as possible new therapeutic option. Epilepsia. 2013 Jan 25. doi: 10.1111/epi.12083. [Epub ahead of print]

    Mellert K, Lamla M, Scheffzek K, Wittig R, Kaufmann D. Enhancing endosomal escape of transduced proteins by photochemical internalisation. PLoS One. 2012;7(12):e52473. doi: 10.1371/journal.pone.0052473. Epub 2012 Dec 21.
    Endo-Porter delivered their test protein (Atto488-BSA) to more cells than other delivery enhancers tested: "Comparing the different transduction reagents, the percentage of fluorescence positive cells varied between 89.2 (TurboFect) and 96.8% (Endo-Porter)."

    Augello MA, Burd CJ, Birbe R, McNair C, Ertel A, Magee MS, Frigo DE, Wilder-Romans K, Shilkrut M, Han S, Jernigan DL, Dean JL, Fatatis A, McDonnell DP, Visakorpi T, Feng FY, Knudsen KE. Convergence of oncogenic and hormone receptor pathways promotes metastatic phenotypes. J Clin Invest. 2013 Jan 2;123(1):493-508. doi: 10.1172/JCI64750. Epub 2012 Dec 21.
    Delivery of Morpholinos into the androgen-dependant LNCaP cancer cell line

    Ridnour LA, Dhanapal S, Hoos M, Wilson J, Lee J, Cheng RY, Brueggemann EE, Hines HB, Wilcock DM, Vitek MP, Wink DA, Colton CA. Nitric oxide-mediated regulation of β-amyloid clearance via alterations of MMP-9/TIMP-1. J Neurochem. 2012 Dec;123(5):736-49. doi: 10.1111/jnc.12028. Epub 2012 Oct 25.
    Delivering to cultured BV2 cells

    Shchelkunova A, Ermolinsky B, Boyle M, Mendez I, Lehker M, Martirosyan KS, Kazansky AV. Tuning of Alternative Splicing - Switch From Proto-Oncogene to Tumor Suppressor. Int J Biol Sci. 2013;9(1):45-54. doi:10.7150/ijbs.5194
    Delivery of Morpholinos into PC-3 cultures

    Wang M, Wu B, Lu P, Cloer C, Tucker JD, Lu Q. Polyethylenimine-modified Pluronics (PCMs) Improve Morpholino Oligomer Delivery in Cell Culture and Dystrophic mdx Mice. Mol Ther. 2012 Nov 20. doi: 10.1038/mt.2012.236. [Epub ahead of print]

    Penn AC, Balik A, Greger IH. Steric antisense inhibition of AMPA receptor Q/R editing reveals tight coupling to intronic editing sites and splicing. Nucleic Acids Res. 2012 Nov 20. [Epub ahead of print]

    Kato Y, Kajiwara C, Ishige I, Mizukami S, Yamazaki C, Eikawa S, Kakimi K, Udono H. HSP70 and HSP90 Differentially Regulate Translocation of Extracellular Antigen to the Cytosol for Cross-Presentation. Autoimmune Dis. 2012;2012:745962. doi: 10.1155/2012/745962. Epub 2012 Sep 25.
    Endo-Porter delivers native and fixed OVA to DC2.4 cells.

    van Dijk M, Thulluru HK, Mulders J, Michel OJ, Poutsma A, Windhorst S, Kleiverda G, Sie D, Lachmeijer AMA, Oudejans CBM. HELLP babies link a novel lincRNA to the trophoblast cell cycle. J Clin Invest. 2012;[Epub ahead of print] doi:10.1172/JCI65171
    Endo-Porter delivery to SGHPL-5 cell line.

    Aburto MR, Sánchez-Calderón H, Hurlé JM, Varela-Nieto I, Magariños M. Early otic development depends on autophagy for apoptotic cell clearance and neural differentiation. Cell Death Dis. 2012 Oct 4;3:e394. doi: 10.1038/cddis.2012.132.
    Endo-Porter delivering Morpholinos to chick inner ear explants.

    Holloway RW, Bogachev O, Bharadwaj AG, McCluskey GD, Majdalawieh AF, Zhang L, Ro HS. Stromal adipocyte enhancer-binding protein 1 (AEBP1) promotes mammary epithelial cell hyperplasia via proinflammatory and hedgehog signaling. J Biol Chem. 2012;[Epub ahead of print] doi:10.1074/jbc.M112.404293

    Yew KH, Carpenter C, Duncan RS, Harrison CJ. Human Cytomegalovirus Induces TLR4 Signaling Components in Monocytes Altering TIRAP, TRAM and Downstream Interferon-Beta and TNF-Alpha Expression. PLoS One. 2012;7(9):e44500. Epub 2012 Sep 7.

    Ridnour LA, Barasch KM, Windhausen AN, Dorsey TH, Lizardo MM, Yfantis HG, Lee DH, Switzer CH, Cheng RYS, Heinecke JL, Brueggmann E, Hines HB, Khanna C, Glynn SA, Ambs S, Wink DA. Nitric Oxide Synthase and Breast Cancer: Role of TIMP-1 in NO-mediated Akt Activation. PLoS ONE. 2012;7(9): e44081. doi:10.1371/journal.pone.0044081
    Delivery to human breast cancer cultures MDA-MB-231.

    Sung HJ, Ma W, Wang PY, Hynes J, O'Riordan TC, Combs CA, McCoy JP Jr, Bunz F, Kang JG, Hwang PM. Mitochondrial respiration protects against oxygen-associated DNA damage. Nat Commun. 2010 Apr 12;1:5. doi: 10.1038/ncomms1003.
    Delivery of an oxygen probe molecule into cultured cells using Endo-Porter.

    Nazmi A, Mukhopadhyay R, Dutta K, Basu A. STING Mediates Neuronal Innate Immune Response Following Japanese Encephalitis Virus Infection. Sci Rep. 2012;2:347. Epub 2012 Apr 2.
    Delivery by Endo-Porter into cultured mouse neuroblastoma Neuro2a (N2a) cells.

    Fang D, Pan C, Lin H, Lin Y, Zhang G, Wang H, He M, Xie L, Zhang R. A novel basic protein, PfN23, functions as a key macromolecule during nacre formation. J Biol Chem. 2012 Mar 13. [Epub ahead of print]
    Morpholino delivery by Endoporter in seawater to oyster embryos.

    Fiszer A, Olejniczak M, Switonski PM, Wroblewska JP, Wisniewska-Kruk JW, Mykowska A, Krzyzosiak WJ. An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases. BMC Mol Biol. 2012;13:6 doi:10.1186/1471-2199-13-6

    Coate TM, Raft S, Zhao X, Ryan AK, Crenshaw EB 3rd, Kelley MW. Otic Mesenchyme Cells Regulate Spiral Ganglion Axon Fasciculation through a Pou3f4/EphA4 Signaling Pathway. Neuron. 2012 Jan 12;73(1):49-63.
    Endo-Porter used with neuron and mesenchyme explants.

    Nakamura K, Du L, Tunuguntla R, Fike F, Cavalieri S, Morio T, Mizutani S, Brusco A, Gatti RA. Functional characterization and targeted correction of ATM mutations identified in Japanese patients with ataxia-telangiectasia. Hum Mutat. 2012 Jan;33(1):198-208. doi: 10.1002/humu.21632. Epub 2011 Nov 9.

    Haririan M, Kaviani S, Soleimani M, Ghaemi SR, Delalat B, Atashi A. Morpholino Oligo Antisense efficiently suppresses BCR/ABL and cell proliferation in CML: specific inhibition of BCR-ABL gene expression by Morpholino Oligo Antisense in BCR-ABL(+) cells. Hematology. 2012 Jan;17(1):28-34.

    Nakano S, Ozasa S, Yoshioka K, Fujii I, Mitsui K, Nomura K, Kosuge H, Endo F, Matsukura M, Kimura S. Exon-skipping events in candidates for clinical trials of morpholino. Pediatr Int. 2011:53(4)524–9.

    Osorio FG, Navarro CL, Cadiñanos J, López-Mejía IC, Quirós PM, Bartoli C, Rivera J, Tazi J, Guzmán G, Varela I, Depetris D, de Carlos F, Cobo J, Andrés V, Sandre-Giovannoli AD, Freije JM, Lévy N, López-Otín C. Splicing-Directed Therapy in a New Mouse Model of Human Accelerated Aging. Sci. Transl. Med. 2011;3:106ra107 doi:10.1126/scitranslmed.3002847

    Zammarchi F, de Stanchina E, Bournazou E, Supakorndej T, Martires K, Riedel E, Corben AD, Bromberg JF, Cartegni L. Antitumorigenic potential of STAT3 alternative splicing modulation. Proc Natl Acad Sci U S A. 2011 Oct 17. [Epub ahead of print]

    McGuire PG, Rangasamy S, Maestas J, Das A. Pericyte-derived sphingosine 1-phosphate induces the expression of adhesion proteins and modulates the retinal endothelial cell barrier. Arterioscler Thromb Vasc Biol. 2011 Dec;31(12):e107-15. Epub 2011 Sep 22.

    Chen S, Bellew C, Yao X, Stefkova J, Dipp S, Saifudeen Z, Bachvarov D, El-Dahr SS. Histone Deacetylase (HDAC) Activity Is Critical for Embryonic Kidney Gene Expression, Growth, and Differentiation. J Biol Chem. 2011 Sep 16;286(37):32775-89. Epub 2011 Jul 21.
    Delivery in murine kidney mK4 cell cultures.

    Lefave CV, Squatrito M, Vorlova S, Rocco GL, Brennan CW, Holland EC, Pan YX, Cartegni L. Splicing factor hnRNPH drives an oncogenic splicing switch in gliomas. EMBO J. 2011 Sep 13;30(19):4084-97. doi: 10.1038/emboj.2011.259.

    Jin Q, Gao G, Mulder KM. Requirement of a dynein light chain in transforming growth factor β signaling in zebrafish ovarian follicle cells. Mol Cell Endocrinol. 2011 Sep 5. [Epub ahead of print]
    293T cultures and zebrafish ovarian follicle cell cultures

    Nazmi A, Dutta K, Basu A. RIG-I Mediates Innate Immune Response in Mouse Neurons Following Japanese Encephalitis Virus Infection. PLoS ONE. 2011;6(6): e21761. doi:10.1371/journal.pone.0021761
    Vivo-Morpholinos for systemic delivery in BALB/c mice, Endo-Porter with bare Morpholinos for delivery to primary cortical neuron culture.

    Wu B, Benrashid E, Lu P, Cloer C, Zillmer A, Shaban M, Lu QL. Targeted Skipping of Human Dystrophin Exons in Transgenic Mouse Model Systemically for Antisense Drug Development. PLoS ONE. 2011;6(5):e19906. doi:10.1371/journal.pone.0019906

    Fernández-Rodríguez J, Castellsagué J, Benito L, Benavente Y, Capellà G, Blanco I, Serra E, Lázaro C. A mild neurofibromatosis type 1 phenotype produced by the combination of the benign nature of a leaky NF1-splice mutation and the presence of a complex mosaicism. Hum Mutat. 2011 Mar 10. doi: 10.1002/humu.21500. [Epub ahead of print]
    Morpholino delivery into fibroblast cultures.

    Rios M, Parada-Bustamante A, Velasquez LA, Croxatto HC, Orihuela PA. Participation of the oviductal s100 calcium binding protein G in the genomic effect of estradiol that accelerates oviductal embryo transport in mated rats. Reproductive Biology and Endocrinology. 2011;9:69 doi:10.1186/1477-7827-9-69
    Delivery in rat oviducts in vivo

    Mutyam V, Puccetti MV, Frisbie J, Goldstein DL, Krane CM. Endo-Porter–mediated delivery of phosphorodiamidate morpholino oligos (PMOs) in erythrocyte suspension cultures from Cope's gray treefrog Hyla chrysoscelis. BioTechniques. 2011 May;50(5)

    Tesz GJ, Aouadi M, Prot M, Nicoloro SM, Boutet E, Amano SU, Goller A, Wang M, Guo CA, Salomon WE, Virbasius JV, Baum RA, O'Connor MJ, Soto E, Ostroff GR, Czech MP. Glucan particles for selective delivery of siRNA to phagocytic cells in mice. Biochem J. 2011 Jun 1;436(2):351-62.

    Koshimizu E, Imamura S, Qi J, Toure J, Valdez DM Jr, Carr CE, Hanai J, Kishi S. Embryonic Senescence and Laminopathies in a Progeroid Zebrafish Model. PLoS ONE. 2011;6(3): e17688. doi:10.1371/journal.pone.0017688
    Delivery into zebrafish AB9 fibroblasts

    Huang W, Ghisletti S, Saijo K, Gandhi M, Aouadi M, Tesz GJ, Zhang DX, Yao J, Czech MP, Goode BL, Rosenfeld MG, Glass CK. Coronin 2A mediates actin-dependent de-repression of inflammatory response genes. Nature. 2011 Feb 17;470(7334):414-8. doi: 10.1038/nature09703.
    Delivery of siRNA using Endo-Porter in β1,3-d-glucan particles, ip injection into mice.

    Bartz R, Fan H, Zhang J, Innocent N, Cherrin C, Beck SC, Pei Y, Momose A, Jadhav V, Tellers DM, Meng F, Crocker LS, Sepp-Lorenzino L, Barnett SF. Effective siRNA delivery and target mRNA degradation using an amphipathic peptide to facilitate pH-dependent endosomal escape. Biochem J. 2011;435:475–87. [Epub Jan 25 ahead of print]
    Tested in HeLa, Huh-7 and Hek293T cultures.

    Hwang-Verslues WW, Chang PH, Wei PC, Yang CY, Huang CK, Kuo WH, Shew JY, Chang KJ, Lee EY, Lee WH. miR-495 is upregulated by E12/E47 in breast cancer stem cells, and promotes oncogenesis and hypoxia resistance via downregulation of E-cadherin and REDD1. Oncogene. 2011 Jan 24. [Epub ahead of print]
    Morpholino delivery to HEK293 cultures.

    Ribeiro CMS, Bird S, Raes G, Ghassabeh GH, Schijns VEJC, Pontes MJSL, Savelkoul HFJ, Wiegertjes GF. A Novel Soluble Immune-Type Receptor (SITR) in Teleost Fish: Carp SITR Is Involved in the Nitric Oxide-Mediated Response to a Protozoan Parasite. PLoS ONE. 2011;6(1): e15986. doi:10.1371/journal.pone.0015986

    Zhu QS, Rosenblatt K, Huang KL, Lahat G, Brobey R, Bolshakov S, Nguyen T, Ding Z, Belousov R, Bill K, Luo X, Lazar A, Dicker A, Mills GB, Hung MC, Lev D. Vimentin is a novel AKT1 target mediating motility and invasion. Oncogene. 2011 Jan 27;30(4):457-70. Epub 2010 Sep 20.
    Morpholinos delivered into primary human STS cells.

    Guan W, Cheng F, Huang Q, Kleiboeker S, Qiu J. Inclusion of the Central Exon of Parvovirus B19 Precursor mRNA is Determined by Multiple Splicing Enhancers both in the Exon and the Downstream Intron. J Virol. 2010 Dec 15. [Epub ahead of print]

    Gong Q, Stump MR, Zhou Z. Inhibition of nonsense-mediated mRNA decay by antisense morpholino oligonucleotides restores functional expression of hERG nonsense and frameshift mutations in long QT syndrome. J Mol Cell Cardiol. 2010 Oct 27. [Epub ahead of print]

    Ghosh G, Subramanian IV, Adhikari N, Zhang X, Joshi HP, Basi D, Chandrashekhar YS, Hall JL, Roy S, Zeng Y, Ramakrishnan S. Hypoxia-induced microRNA-424 expression in human endothelial cells regulates HIF-α isoforms and promotes angiogenesis. J Clin Invest. 2010 Oct 25. pii: 42980. doi: 10.1172/JCI42980. [Epub ahead of print]
    Morpholino delivery into HUVEC cultures

    Li YH, Chen MH, Gong HY, Hu SY, Li YW, Lin GH, Lin CC, Liu W, Wu JL. Progranulin A-mediated MET signaling is essential for liver morphogenesis in zebrafish. J Biol Chem. 2010 Oct 20. [Epub ahead of print]
    Morpholino delivery into ZFL cultures (zebafish liver culture)

    Zhang J, Harrison JS, Studzinski GP. Isoforms of p38MAPK gamma and delta contribute to differentiation of human AML cells induced by 1,25-dihydroxyvitamin D₃. Exp Cell Res. 2011 Jan 1;317(1):117-30. Epub 2010 Sep 8.
    Endo-Porter delivering phosphorothioated antisense chimeras.

    Zhdanov AV, Dmitriev RI, Papkovsky DB. Bafilomycin A1 activates respiration of neuronal cells via uncoupling associated with flickering depolarization of mitochondria. Cell Mol Life Sci. 2011 March; 68(5): 903–917. Published online 2010 September 6. doi: 10.1007/s00018-010-0502-8.

    de Campos-Nebel M, Larripa I, González-Cid M. Topoisomerase II-Mediated DNA Damage Is Differently Repaired during the Cell Cycle by Non-Homologous End Joining and Homologous Recombination. PLoS One. 2010 Sep 2;5(9). pii: e12541.

    Saito T, Nakamura A, Aoki Y, Yokota T, Okada T, Osawa M, Takeda S. Antisense PMO Found in Dystrophic Dog Model Was Effective in Cells from Exon 7-Deleted DMD Patient. PLoS ONE. 2010 Aug;5(8):e12239. doi:10.1371/journal.pone.0012239

    Ghigna C, De Toledo M, Bonomi S, Valacca C, Gallo S, Apicella M, Eperon I, Tazi J, Biamonti G. Pro-metastatic splicing of Ron proto-oncogene mRNA can be reversed: therapeutic potential of bifunctional oligonucleotides and indole derivatives. RNA Biol. 2010 Jul-Aug;7(4):495-503. Epub 2010 Jul 1.

    Song R, Van Buren T, Yosypiv IV. Histone deacetylases are critical regulators of the renin-angiotensin system during ureteric bud branching morphogenesis. Pediatr Res. 2010 Jun;67(6):573-8.
    mouse MK4 embryonic metanephric mesenchyme cell line

    Long J, Wang Y, Wang W, Chang BH, Danesh FR. Identification of MicroRNA-93 as a novel regulator of vascular endothelial growth factor (VEGF) In hyperglycemic conditions. J Biol Chem. 2010 May 25. [Epub ahead of print]
    Abstract mentions peptide-conjugated Morpholino oligomers, but mat'l and methods describes Endo-Porter.

    Martinon F, Chen X, Lee AH, Glimcher LH. TLR activation of the transcription factor XBP1 regulates innate immune responses in macrophages. Nat Immunol. 2010 May;11(5):411-8. Epub 2010 Mar 28.
    Endo-Porter used to deliver an active recombinant IRE1α protein fragment into J774 macrophages.

    Lahat G, Zhu QS, Huang KL, Wang S, Bolshakov S, Liu J, Torres K, Langley RR, Lazar AJ, Hung MC, Lev D. Vimentin is a novel anti-cancer therapeutic target; insights from in vitro and in vivo mice xenograft studies. PLoS One. 2010 Apr 16;5(4):e10105.

    Matluk N, Rochiera JA, Karaczyn A, Adams TL, Verdi JM. A role for NRAGE in NF-kappaB activation through the non-canonical BMP pathway. BMC Biol. 2010 Jan 25;8(1):7. [Epub ahead of print]

    Pillai VC, Yesudas R, Shaik IH, Thekkumkara TJ, Bickel U, Srivenugopal KS, Mehvar R. Delivery of NADPH-Cytochrome P450 Reductase Antisense Oligos Using Avidin-Biotin Approach. Bioconjug Chem. 2010 Jan 11. [Epub ahead of print]

    Ikeuchi N, Futami J, Hosoi A, Noji S, Kurachi M, Ueha S, Fujii SI, Yamada H, Matsushima K, Moriyasu F, Kakimi K. Efficient cross-presentation of soluble exogenous antigens introduced into dendritic cells using a weak-based amphiphilic peptide. Biochem Biophys Res Commun. 2010 Jan 11. [Epub ahead of print]

    Fercher A, Ponomarev GV, Yashunski D, Papkovsky D. Evaluation of the derivates of phosphorescent Pt-coproporphyrin as intracellular oxygen-sensitive probes. Anal Bioanal Chem. 2010 Jan 10. [Epub ahead of print]

    Spigoni G, Gedressi C, Mallamaci A. Regulation of Emx2 expression by antisense transcripts in murine cortico-cerebral precursors. PLoS One. 2010 Jan 11;5(1):e8658.
    Morpholinos were delivered to NIH/3T3 cells.

    Figeac F, Uzan B, Faro M, Chelali N, Portha B, Movassat J. Neonatal growth and regeneration of {beta} cells are regulated by the Wnt/{beta}-catenin signaling in normal and diabetic rats. Am J Physiol Endocrinol Metab. 2009 Nov 17. [Epub ahead of print]

    Yew KH, Carsten B, Harrison C. Scavenger receptor A1 is required for sensing HCMV by endosomal TLR-3/-9 in monocytic THP-1 cells. Mol Immunol. 2009 Nov 13. [Epub ahead of print]

    Nossa CW, Jain P, Tamilselvam B, Gupta VR, Chen LF, Schreiber V, Desnoyers S, Blanke SR. Activation of the abundant nuclear factor poly(ADP-ribose) polymerase-1 by Helicobacter pylori. Proc Natl Acad Sci U S A. 2009 Nov 24;106(47):19998-20003. Epub 2009 Nov 6.

    Chen AY, Zhang EY, Guan W, Cheng F, Kleiboeker S, Yankee TM, Qiu J. The small 11kDa non-structural protein of human parvovirus B19 plays a key role in inducing apoptosis during B19 virus infection of primary erythroid progenitor cells. Blood. 2009 Oct 27. [Epub ahead of print]

    Shieh JJ, Liu KT, Huang SW, Chen YJ, Hsieh TY. Modification of Alternative Splicing of Mcl-1 Pre-mRNA Using Antisense Morpholino Oligonucleotides Induces Apoptosis in Basal Cell Carcinoma Cells. J Invest Dermatol. 2009 Oct;129(10):2497-506. doi: 10.1038/jid.2009.83. Epub 2009 Apr 16.

    Maxhimer JB, Soto-Pantoja DR, Ridnour LA, Shih HB, DeGraff WG, Tsokos M, Wink DA, Isenberg JS, Roberts DD. Radioprotection in Normal Tissue and Delayed Tumor Growth by Blockade of CD47 Signaling. Sci Transl Med. 2009;1(3):3ra7

    Pérez B, Rincón A, Jorge-Finnigan A, Richard E, Merinero B, Ugarte M, Desviat LR. Pseudoexon exclusion by antisense therapy in methylmalonic aciduria (MMAuria). Hum Mutat. 2009 Sep 2. [Epub ahead of print]

    Okamoto M, Van Stry M, Chung L, Koyanagi M, Sun X, Suzuki Y, Ohara O, Kitamura H, Hijikata A, Kubo M, Bix M. Mina, an Il4 repressor, controls T helper type 2 bias. Nat Immunol. 2009 Aug;10(8):872-9. Epub 2009 Jun 28.
    Primary C57BL/6 CD4+ T cells

    Rodríguez-Pascau L, Coll MJ, Vilageliu L, Grinberg D. Antisense oligonucleotide treatment for a pseudoexon-generating mutation in the NPC1 gene causing Niemann-Pick type C disease. Hum Mutat. 2009 Aug 30. [Epub ahead of print]

    Jenny MJ, Karchner S, Franks D, Woodin BW, Stegeman JJ, Hahn ME. Distinct roles of two zebrafish AHR repressors (AHRRa and AHRRb) in embryonic development and regulating the response to TCDD (2,3,7,8-tetrachlorodibenzo-p-dioxin). Toxicol Sci. 2009 Jun 3. [Epub ahead of print]
    Delivery into zebrafish cell line ZF-L.

    Lin Z, Murtaza I, Wang K, Jiao J, Gao J, Li PF. miR-23a functions downstream of NFATc3 to regulate cardiac hypertrophy. Proc Natl Acad Sci U S A. 2009 Jul 2. [Epub ahead of print]

    Liu B, Paranjpe S, Bowen WC, Bell AW, Luo JH, Yu YP, Mars WM, Michalopoulos GK. Investigation of the Role of Glypican 3 in Liver Regeneration and Hepatocyte Proliferation. Am J Pathol. 2009 Jul 2. [Epub ahead of print]

    Qi YT, Zhao Y, Zhang Z, Li D, Gu JY, Fu XN, Chen XN, Yuan CG, Lai LH. A study of mir-34a effect on expression and function in nerves. Journal of Molecular Cell Biology. 2009;(42)3-4:217-223 (Chinese)

    Nikopoulos GN, Martins JF, Adams TL, Karaczyn A, Adams D, Vary C, Verdi JM. NRAGE: A Potential Rheostat During Branching Morphogenesis. Mech Dev. 2009 May-Jun;126(5-6):337-49. Epub 2009 Mar 4.

    Kamel S, Kruger C, Salbaum JM, Kappen C. Morpholino-mediated knockdown in primary chondrocytes implicates Hoxc8 in regulation of cell cycle progression. Bone. 2009 Apr;44(4):708-16. doi: 10.1016/j.bone.2008.10.057. Epub 2008 Nov 21.

    Aouadi M, Tesz GJ, Nicoloro SM, Wang M, Chouinard M, Soto E, Ostroff GR, Czech MP. Orally delivered siRNA targeting macrophage Map4k4 suppresses systemic inflammation. Nature. 2009 Apr 30;458(7242):1180-4.

    Coate TM, Swanson TL, Copenhaver PF. Reverse signaling by glycosylphosphatidylinositol-linked manduca ephrin requires a SRC family kinase to restrict neuronal migration in vivo. J Neurosci. 2009 Mar 18;29(11):3404-18.

    Wang X, Gocek E, Liu CG, Studzinski GP. MicroRNAs181 regulate the expression of p27Kip1 in human myeloid leukemia cells induced to differentiate by 1,25-dihydroxyvitamin D3. Cell Cycle. 2009 Mar 1;8(5):736-41. Epub 2009 Mar 18.
    MicroRNA precursors (pre-miRNA) delivered into HL60 and U937 cells.

    Uzan B, Figeac F, Portha B, Movassat J. Mechanisms of KGF Mediated Signaling in Pancreatic Duct Cell Proliferation and Differentiation. PLoS ONE. 2009;4(3):e4734. Epub 2009 Mar 6.

    Pros E, Fernández-Rodríguez J, Canet B, Benito L, Sánchez A, Benavides A, Ramos FJ, López-Ariztegui MA, Capellá G, Blanco I, Serra E, Lázaro C. Antisense therapeutics for neurofibromatosis type 1 caused by deep intronic mutations. Hum Mutat. 2009 Feb 24;30(3):454-462. [Epub ahead of print]

    O'Hagan KA, Cocchiglia S, Zhdanov AV, Tambawala MM, Cummins EP, Monfared M, Agbor TA, Garvey JF, Papkovsky DB, Taylor CT, Allan BB. PGC-1alpha is coupled to HIF-1alpha-dependent gene expression by increasing mitochondrial oxygen consumption in skeletal muscle cells. Proc Natl Acad Sci U S A. 2009 Feb 17;106(7):2188-93. Epub 2009 Jan 28.

    Göoz P, Göoz M, Baldys A, Hoffman S. ADAM-17 regulates endothelial cell morphology, proliferation, and in vitro angiogenesis. Biochem Biophys Res Commun. 2009 Feb 27;380(1):33-8. Epub 2009 Jan 14.

    Lynch VJ, Tanzer A, Wang Y, Leung FC, Gellersen B, Emera D, Wagner GP. Adaptive changes in the transcription factor HoxA-11 are essential for the evolution of pregnancy in mammals. Proc Natl Acad Sci U S A. 2008 Sep 30;105(39):14928-33. Epub 2008 Sep 22.

    Davis RL, Homer VM, George PM, Brennan SO. A deep intronic mutation in FGB creates a consensus exonic splicing enhancer motif that results in afibrinogenemia caused by aberrant mRNA splicing, which can be corrected in vitro with antisense oligonucleotide treatment. Hum Mutat. 2008 Oct 13. [Epub ahead of print]

    Sun CS, Wu KT, Lee HH, Uen YH, Tian YF, Tzeng CC, Wang AH, Cheng CJ, Tsai SL. Anti-sense morpholino oligonucleotide assay shows critical involvement for NF-kappaB activation in the production of Wnt-1 protein by HepG2 cells: oncology implications. J Biomed Sci. 2008 Sep;15(5):633-43. Epub 2008 May 7.

    Staszkiewicz J, Gimble J, Manuel JA, Gawronska-Kozak B. IFATS Series: Stem Cell Antigen-1 Positive Ear Mesenchymal Stem Cells (EMSC) Display Enhanced Adipogenic Potential. Stem Cells. 2008 Jul 3. [Epub ahead of print] Morpholinos were delivered into C57BL/6J mouse ear mesenchymal stem cells.

    Chiu SL, Chen CM, Cline HT. Insulin receptor signaling regulates synapse number, dendritic plasticity, and circuit function in vivo. Neuron. 2008 Jun 12;58(5):708-19.
    Morpholinos delivered into HEK293 cells.

    Kahler RA, Yingst SM, Hoeppner LH, Jensen ED, Krawczak D, Oxford JT, Westendorf JJ. Collagen 11a1 is indirectly activated by lymphocyte enhancer-binding factor 1 (Lef1) and negatively regulates osteoblast maturation. Matrix Biol. 2008 May;27(4):330-8. Epub 2008 Jan 16.

    Isenberg JS, Romeo MJ, Maxhimer JB, Smedley J, Frazier WA, Roberts DD. Gene Silencing of CD47 and Antibody Ligation of Thrombospondin-1 Enhance Ischemic Tissue Survival in a Porcine Model: Implications for Human Disease. Annals of Surgery. 2008 May;247(5):860-868.

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