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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 11708 scientific papers returned from the database with the search filters currently being used below.

In Vivo Evaluation of Exon 51 Skipping in hDMD/Dmd-null Mice

Authors:
Sheri N, Yokota T
Citation:
Methods Mol Biol. 2023;2640:327-336. doi: 10.1007/978-1-0716-3036-5_23. PMID: 36995605
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is a fatal X-linked condition that affects 1 in 3500-6000 newborn boys a year. An out-of-...
Link:
https://link.springer.com/protocol/10.1007/978-1-0716-3036-5_23
Delivery Method:
intravenous (i.v.) and intramuscular (i.m.) injections
Organism or Cell Type:
mice
Citation Extract:
Sheri N, Yokota T. In Vivo Evaluation of Exon 51 Skipping in hDMD/Dmd-null Mice. Methods Mol Biol. 2023;2640:327-336. doi: 10.1007/978-1-0716-3036-5_23. PMID: 36995605.

Exons 45-55 Skipping Using Antisense Oligonucleotides in Immortalized Human DMD Muscle Cells

Authors:
He M, Yokota T
Citation:
Methods Mol Biol. 2023;2640:313-325. doi: 10.1007/978-1-0716-3036-5_22.
Epub:
Not Epub
Abstract:
Antisense oligonucleotides (AOs) have demonstrated high potential as a therapy for treating genetic diseases like Duchene...
Link:
https://link.springer.com/protocol/10.1007/978-1-0716-3036-5_22
Organism or Cell Type:
cell culture: immortalized DMD muscle cell lines
Citation Extract:
He M, Yokota T. Exons 45-55 Skipping Using Antisense Oligonucleotides in Immortalized Human DMD Muscle Cells. Methods Mol Biol. 2023;2640:313-325. doi: 10.1007/978-1-0716-3036-5_22..

Improved Prediction of Antisense Oligonucleotide Efficacy for Exon Skipping Using Ensemble Learning and Feature Selection

Authors:
Zhu A, Chiba S, Shimizu Y, Kunitake K, Okuno Y, Aoki Y, Yokota T
Citation:
Preprints. 2023;2023030389. doi:10.20944/preprints202303.0389.v1
Epub:
Not Epub
Abstract:
Antisense oligonucleotide (ASO)-mediated exon skipping has become a valuable tool for investigating gene function and...
Link:
https://www.preprints.org/manuscript/202303.0389/v1
Citation Extract:
Zhu A, Chiba S, Shimizu Y, Kunitake K, Okuno Y, Aoki Y, Yokota T. Improved Prediction of Antisense Oligonucleotide Efficacy for Exon Skipping Using Ensemble Learning and Feature Selection. Preprints. 2023;2023030389. doi:10.20944/preprints202303.0389.v1.

ASO targeting RBM3 temperature-controlled poison exon splicing prevents neurodegeneration in vivo

Authors:
Preußner M, Smith HL, Hughes D, Zhang M, Emmerichs AK, Scalzitti S, Peretti D, Swinden D, Neumann A, Haltenhof T, Mallucci GR, Heyd F
Citation:
EMBO Mol Med. 2023 Mar 22:e17157. doi: 10.15252/emmm.202217157. Online ahead of print
Epub:
Yes
Abstract:
Neurodegenerative diseases are increasingly prevalent in the aging population, yet no disease-modifying treatments are...
Link:
https://www.embopress.org/doi/full/10.15252/emmm.202217157
Delivery Method:
Endo-Porter
Organism or Cell Type:
cell culture: HEK293
Citation Extract:
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.

Exosome-derived circCCAR1 promotes CD8 + T-cell dysfunction and anti-PD1 resistance in hepatocellular carcinoma

Authors:
Hu Z, Chen G, Zhao Y, Gao H, Li L, Yin Y, Jiang J, Wang L, Mang Y, Gao Y, Zhang S, Ran J, Li L
Citation:
Mol Cancer. 2023 Mar 18;22(1):55. doi:10.1186/s12943-023-01759-1
Epub:
Not Epub
Abstract:
Background: Circular RNAs (circRNAs) can be encapsulated into exosomes to participate in intercellular communication, affecting...
Link:
https://molecular-cancer.biomedcentral.com/articles/10.1186/s12943-023-01759-1
Organism or Cell Type:
cell culture: primary human hepatocellular carcinoma (HCC)
Citation Extract:
Hu Z, Chen G, Zhao Y, Gao H, Li L, Yin Y, Jiang J, Wang L, Mang Y, Gao Y, Zhang S, Ran J, Li L. Exosome-derived circCCAR1 promotes CD8 + T-cell dysfunction and anti-PD1 resistance in hepatocellular carcinoma. Mol Cancer. 2023 Mar 18;22(1):55. doi:10.1186/s12943-023-01759-1.

Isoform-specific knockdown of long and intermediate prolactin receptors interferes with evolution of B-cell neoplasms

Authors:
Taghi Khani A, Kumar A, Sanchez Ortiz A, Radecki C, Aramburo S, Lee SJ, Hu Z, Damirchi B,Lorenson MY, Wu X, Gu Z, Stohl W, Sanz I, Meffre E, Müschen M, Forman SJ, Koff JL, Walker AM. Swaminathan S
Citation:
Commun Biol. 2023;6:295. doi:10.1038/s42003-023-04667-8
Epub:
Not Epub
Abstract:
Prolactin (PRL) is elevated in B-cell-mediated lymphoproliferative diseases and promotes B-cell survival. Whether PRL or PRL...
Link:
https://www.nature.com/articles/s42003-023-04667-8
Delivery Method:
Alzet osmotic pump Vivo-Morpholino
Organism or Cell Type:
NOD-SCID IL2Rγ−/− (NSG) mice xenografted
Citation Extract:
Taghi Khani A, Kumar A, Sanchez Ortiz A, Radecki C, Aramburo S, Lee SJ, Hu Z, Damirchi B,Lorenson MY, Wu X, Gu Z, Stohl W, Sanz I, Meffre E, Müschen M, Forman SJ, Koff JL, Walker AM. Swaminathan S. Isoform-specific knockdown of long and intermediate prolactin receptors interferes with evolution of B-cell neoplasms. Commun Biol. 2023;6:295. doi:10.1038/s42003-023-04667-8.

Variations in the poly-histidine repeat motif of HOXA1 contribute to bicuspid aortic valve in mouse and zebrafish

Authors:
Odelin G, Faucherre A, Marchese D, Pinard A, Jaouadi H, Le Scouarnec S, Deleuze J-F, Génin E, Lindenbaum P, Redon R, Schott J-J, Chiarelli R, Achouri Y, Faure E, Herbane M, Théron A, Avierinos J-F, Jopling C, Collod-Béroud G, Rezsohazy R, Zaffran S
Citation:
Nat Commun. 2023;14,1543. doi:10.1038/s41467-023-37110-x
Epub:
Not Epub
Abstract:
Bicuspid aortic valve (BAV), the most common cardiovascular malformation occurs in 0.5-1.2% of the population. Although highly...
Link:
https://www.nature.com/articles/s41467-023-37110-x
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Odelin G, Faucherre A, Marchese D, Pinard A, Jaouadi H, Le Scouarnec S, Deleuze J-F, Génin E, Lindenbaum P, Redon R, Schott J-J, Chiarelli R, Achouri Y, Faure E, Herbane M, Théron A, Avierinos J-F, Jopling C, Collod-Béroud G, Rezsohazy R, Zaffran S. Variations in the poly-histidine repeat motif of HOXA1 contribute to bicuspid aortic valve in mouse and zebrafish. Nat Commun. 2023;14,1543. doi:10.1038/s41467-023-37110-x.

CLN3 deficiency leads to neurological and metabolic perturbations during early development

Authors:
Heins-Marroquin U, Singh RR, Perathoner S, Gavotto F, Ruiz CM, Patraskaki M, Gomez-Giro G, Borgmann FK, Meyer M, Carpentier A, Warmoes MO, Jaeger C, Mittelbronn M, Schwamborn JC, Cordero-Maldonado ML, Crawford AD, Schymanski EL, Linster CL
Citation:
bioRxiv. 2023;[preprint] doi:10.1101/2023.03.17.533107
Epub:
Not Epub
Abstract:
Juvenile Neuronal Ceroid Lipofuscinosis (or Batten disease) is an autosomal recessive, rare neurodegenerative disorder that...
Link:
https://www.biorxiv.org/content/10.1101/2023.03.17.533107v1
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Heins-Marroquin U, Singh RR, Perathoner S, Gavotto F, Ruiz CM, Patraskaki M, Gomez-Giro G, Borgmann FK, Meyer M, Carpentier A, Warmoes MO, Jaeger C, Mittelbronn M, Schwamborn JC, Cordero-Maldonado ML, Crawford AD, Schymanski EL, Linster CL. CLN3 deficiency leads to neurological and metabolic perturbations during early development. bioRxiv. 2023;[preprint] doi:10.1101/2023.03.17.533107.

Self-transfecting GMO-PMO antisense chimera targeting Nanog enable gene silencing in vitro and suppresses tumor growth in 4T1 allografts in mouse

Authors:
Das U, Kundu J, Shaw P, Bose C, Ghosh A, Gupta S, Sarkar S, Bhadra J, Sinha S
Citation:
Mol Ther Nucleic Acids. 2023;[Epub] doi:10.1016/j.omtn.2023.03.011
Epub:
Yes
Abstract:
Phosphorodiamidate Morpholino Oligonucleotides (PMOs)-based antisense reagents cannot enter cells without help of any delivery...
Link:
https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(23)00070-7
Delivery Method:
GMO-PMO
Organism or Cell Type:
cell culture: MCF7; zebrafish; BALB/c mice
Citation Extract:
Das U, Kundu J, Shaw P, Bose C, Ghosh A, Gupta S, Sarkar S, Bhadra J, Sinha S. Self-transfecting GMO-PMO antisense chimera targeting Nanog enable gene silencing in vitro and suppresses tumor growth in 4T1 allografts in mouse. Mol Ther Nucleic Acids. 2023;[Epub] doi:10.1016/j.omtn.2023.03.011.

Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models

Authors:
Saoudi A, Fergus C, Gileadi T, Montanaro F, Morgan JE, Kelly VP, Tensorer T, Garcia L, Vaillend C, Muntoni F, Goyenvalle A
Citation:
Cells. 2023;12(6):908. doi:10.3390/cells12060908
Epub:
Not Epub
Abstract:
Nucleic acid-based therapies have demonstrated great potential for the treatment of monogenetic diseases, including neurologic...
Link:
https://www.mdpi.com/2073-4409/12/6/908
Delivery Method:
intraperitoneal ip, intracerebroventricular icv, intra-cisterna magna icm, osmotic pump
Organism or Cell Type:
mice
Citation Extract:
Saoudi A, Fergus C, Gileadi T, Montanaro F, Morgan JE, Kelly VP, Tensorer T, Garcia L, Vaillend C, Muntoni F, Goyenvalle A. Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models. Cells. 2023;12(6):908. doi:10.3390/cells12060908.

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