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Morpholino Publication Database

This database contains citations and abstracts for research using Morpholino oligos, as well as some review articles incorporating Morpholino data. You can search the content using the filter boxes below.

There are 268 scientific papers returned from the database with the search filters currently being used below.

Hexose potentiates peptide-conjugated morpholino oligomer efficacy in cardiac muscles of dystrophic mice in an age-dependent manner

Authors:
Han G, Gu B, Lin C, Ning H, Song J, Gao X, Moulton HM, Yin HF
Citation:
Mol Ther Nucl Acids. 2019 Sep 23;18:341-350. doi: 10.1016/j.omtn.2019.09.012. [Epub ahead of print]
Epub:
Not Epub
Abstract:
Insufficient delivery of oligonucleotides to muscle and heart remains a barrier for clinical implementation of antisense...
Link:
http://www.sciencedirect.com/science/article/pii/S2162253119302598
Delivery Method:
peptide-linked
Organism or Cell Type:
mice mdx
Citation Extract:
Han G, Gu B, Lin C, Ning H, Song J, Gao X, Moulton HM, Yin HF. Hexose potentiates peptide-conjugated morpholino oligomer efficacy in cardiac muscles of dystrophic mice in an age-dependent manner. Mol Ther Nucl Acids. 2019 Sep 23;18:341-350. doi: 10.1016/j.omtn.2019.09.012. [Epub ahead of print].

Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides

Authors:
Aung-Htut MT, Comerford I, Johnsen R, Foyle K, Fletcher S, Wilton SD
Citation:
Sci Rep. 2019;9(1):12994. doi:10.1038/s41598-019-49385-6
Epub:
Not Epub
Abstract:
With recent approvals of antisense oligonucleotides as therapeutics, there is an increasing interest in expanding the...
Link:
https://www.nature.com/articles/s41598-019-49385-6
Delivery Method:
in vitro lipofectamine (fibroblasts) electroporation (Jurkat) , in vivo peptide-linked
Organism or Cell Type:
cell culture: primary dermal fibroblasts, Jurkat; autoimmune encephalomyelitis mouse model of multiple sclerosis
Citation Extract:
Aung-Htut MT, Comerford I, Johnsen R, Foyle K, Fletcher S, Wilton SD. Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides. Sci Rep. 2019;9(1):12994. doi:10.1038/s41598-019-49385-6.

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice

Authors:
Klein AF, Varela MA, Arandel L, Holland A, Naouar N, Arzumanov A, Seoane D, Revillod L, Bassez G, Ferry A, Jauvin D, Gourdon G, Puymirat J, Gait MJ, Furling D, Wood MJA
Citation:
J Clin Invest. 2019 Nov 1;129(11):4739-4744. doi: 10.1172/JCI128205
Epub:
Not Epub
Abstract:
Antisense oligonucleotides (ASOs) targeting pathologic RNAs have shown promising therapeutic corrections for many genetic...
Link:
https://www.jci.org/articles/view/128205
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: patient-derived muscle DM1 mice
Citation Extract:
Klein AF, Varela MA, Arandel L, Holland A, Naouar N, Arzumanov A, Seoane D, Revillod L, Bassez G, Ferry A, Jauvin D, Gourdon G, Puymirat J, Gait MJ, Furling D, Wood MJA. Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice. J Clin Invest. 2019 Nov 1;129(11):4739-4744. doi: 10.1172/JCI128205.

Chimeras of Cell-Penetrating Peptides Demonstrate Synergistic Improvement in Antisense Efficacy

Authors:
Fadzen CM, Holden R, Wolfe JM, Choo Z-N, Schissel CK, Yao M, Hanson GJ, Pentelute BL
Citation:
Biochemistry. 2019;[Epub ahead of print] doi:10.1021/acs.biochem.9b00413
Epub:
Yes
Abstract:
Phosphorodiamidate morpholino oligonucleotides (PMOs) are a promising class of therapeutics for genetic disease. PMOs designed...
Link:
https://pubs.acs.org/doi/pdf/10.1021/acs.biochem.9b00413
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: HeLa-654
Citation Extract:
Fadzen CM, Holden R, Wolfe JM, Choo Z-N, Schissel CK, Yao M, Hanson GJ, Pentelute BL. Chimeras of Cell-Penetrating Peptides Demonstrate Synergistic Improvement in Antisense Efficacy. Biochemistry. 2019;[Epub ahead of print] doi:10.1021/acs.biochem.9b00413.

TMPRSS2 is the major activating protease of influenza A virus in primary human airway cells and influenza B virus in human type II pneumocytes

Authors:
Limburg H, Harbig A, Bestle D, Stein DA, Moulton HM, Jaeger J, Janga H, Hardes K, Koepke J, Schulte L, Koczulla AR, Schmeck B, Klenk HD, Böttcher-Friebertshäuser E
Citation:
J Virol. 2019 Oct 15;93(21):e00649-19. doi: 10.1128/JVI.00649-19. PMID: 31391268; PMCID: PMC6803253
Epub:
Yes
Abstract:
Cleavage of influenza virus hemagglutinin (HA) by host cell proteases is essential for virus infectivity and spread. We...
Link:
https://journals.asm.org/doi/10.1128/jvi.00649-19
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: primary human bronchial epithelial cells (HBEC), primary type II alveolar epithelial cells (AECII) and Calu-3 cells
Citation Extract:
Limburg H, Harbig A, Bestle D, Stein DA, Moulton HM, Jaeger J, Janga H, Hardes K, Koepke J, Schulte L, Koczulla AR, Schmeck B, Klenk HD, Böttcher-Friebertshäuser E. TMPRSS2 is the major activating protease of influenza A virus in primary human airway cells and influenza B virus in human type II pneumocytes. J Virol. 2019 Oct 15;93(21):e00649-19. doi: 10.1128/JVI.00649-19. PMID: 31391268; PMCID: PMC6803253.

Global analysis of polysome-associated mRNA in vesicular stomatitis virus infected cells

Authors:
Neidermyer WJ Jr, Whelan SPJ
Citation:
PLoS Pathog. 2019;15(6):e1007875. doi:10.1371/journal.ppat.1007875
Epub:
Not Epub
Abstract:
Infection of mammalian cells with vesicular stomatitis virus (VSV) results in the inhibition of cellular translation while...
Link:
https://journals.plos.org/plospathogens/article?id=10.1371/journal.ppat.1007875
Delivery Method:
peptide-linked
Organism or Cell Type:
cell culture: HeLa
Citation Extract:
Neidermyer WJ Jr, Whelan SPJ. Global analysis of polysome-associated mRNA in vesicular stomatitis virus infected cells. PLoS Pathog. 2019;15(6):e1007875. doi:10.1371/journal.ppat.1007875.

AcrAB-TolC Inhibition by Peptide-conjugated Phosphorodiamidate Morpholino Oligomers Restores Antibiotic Activity in vitro and in vivo

Authors:
Sturge CR, Felder-Scott CF, Pifer R, Pybus C, Jain R, Geller B, Greenberg D
Citation:
ACS Infect Dis. 2019 May 23. doi: 10.1021/acsinfecdis.9b00123. [Epub ahead of print]
Epub:
Yes
Abstract:
Overexpression of bacterial efflux pumps are a driver of increasing antibiotic resistance in gram-negative pathogens. The AcrAB...
Link:
https://pubs.acs.org/doi/10.1021/acsinfecdis.9b00123
Delivery Method:
peptide-linked
Organism or Cell Type:
Enterobacteriaceae strains, Klebsiella pneumoniae, Pseudomonas aeruginosa
Citation Extract:
Sturge CR, Felder-Scott CF, Pifer R, Pybus C, Jain R, Geller B, Greenberg D. AcrAB-TolC Inhibition by Peptide-conjugated Phosphorodiamidate Morpholino Oligomers Restores Antibiotic Activity in vitro and in vivo. ACS Infect Dis. 2019 May 23. doi: 10.1021/acsinfecdis.9b00123. [Epub ahead of print].

Scavenger receptor class A1 mediates uptake of morpholino antisense oligonucleotide into dystrophic skeletal muscle

Authors:
Miyatake S, Mizobe Y, Tsoumpra M, Lim KRQ, Hara Y, Shabanpoor F, Yokota T, Takeda S, Aoki Y
Citation:
Mol Ther Nucl Acids. 2019 Jan 25;14:520-535. doi: 10.1016/j.omtn.2019.01.008.
Epub:
Not Epub
Abstract:
Exon-skipping using phosphorodiamidate morpholino oligomers (PMOs) is a promising treatment strategy for Duchenne muscular...
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(19)30012-5
Delivery Method:
peptide-linked, cell bathing
Organism or Cell Type:
mice mdx62, cell culture: H2K-mdx 52 myoblasts
Citation Extract:
Miyatake S, Mizobe Y, Tsoumpra M, Lim KRQ, Hara Y, Shabanpoor F, Yokota T, Takeda S, Aoki Y. Scavenger receptor class A1 mediates uptake of morpholino antisense oligonucleotide into dystrophic skeletal muscle. Mol Ther Nucl Acids. 2019 Jan 25;14:520-535. doi: 10.1016/j.omtn.2019.01.008. .

Comprehensive RNA-Sequencing Analysis in Serum and Muscle Reveals Novel Small RNA Signatures with Biomarker Potential for DMD

Authors:
Coenen-Stass AML, Sork H, Gatto S, Godfrey C, Bhomra A, Krjutškov K, Hart JR, Westholm JO, O'Donovan L, Roos A, Lochmüller H, Puri PL, El Andaloussi S, Wood MJA, Roberts TC
Citation:
Mol Ther Nucleic Acids. 2018 Dec 7;13:1-15. doi: 10.1016/j.omtn.2018.08.005. Epub 2018 Aug 17
Epub:
Not Epub
Abstract:
Extracellular small RNAs (sRNAs), including microRNAs (miRNAs), are promising biomarkers for diseases such as Duchenne muscular...
Link:
https://www.sciencedirect.com/science/article/pii/S2162253118302105
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Coenen-Stass AML, Sork H, Gatto S, Godfrey C, Bhomra A, Krjutškov K, Hart JR, Westholm JO, O'Donovan L, Roos A, Lochmüller H, Puri PL, El Andaloussi S, Wood MJA, Roberts TC. Comprehensive RNA-Sequencing Analysis in Serum and Muscle Reveals Novel Small RNA Signatures with Biomarker Potential for DMD. Mol Ther Nucleic Acids. 2018 Dec 7;13:1-15. doi: 10.1016/j.omtn.2018.08.005. Epub 2018 Aug 17.

Embryonic myosin is a regeneration marker to monitor utrophin based therapies for DMD

Authors:
Guiraud S, Edwards B, Squire SE, Moir L, Berg A, Babbs A, Ramadan N, Wood MJ, Davies KE
Citation:
Hum Mol Genet. 2018;ddy353 doi:10.1093/hmg/ddy353
Epub:
Yes
Abstract:
Duchenne muscular dystrophy (DMD) is a lethal, X-linked muscle-wasting disease caused by lack of the cytoskeletal protein...
Link:
https://academic.oup.com/hmg/advance-article/doi/10.1093/hmg/ddy353/5124574
Delivery Method:
peptide-linked
Organism or Cell Type:
mice mdx
Citation Extract:
Guiraud S, Edwards B, Squire SE, Moir L, Berg A, Babbs A, Ramadan N, Wood MJ, Davies KE. Embryonic myosin is a regeneration marker to monitor utrophin based therapies for DMD. Hum Mol Genet. 2018;ddy353 doi:10.1093/hmg/ddy353 .

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