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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 11661 scientific papers returned from the database with the search filters currently being used below.

Developing Therapeutic Splice-Correcting Antisense Oligomers for Adult-Onset Pompe Disease with c.-32-13T>G Mutation

Authors:
Ham KA, Johnsen RD, Tchan M, Wilton SD, Aung-Htut MT
Citation:
Methods Mol Biol. 2023;2587:239-251. doi: 10.1007/978-1-0716-2772-3_14
Epub:
Not Epub
Abstract:
The mutation c.-32-13T>G in the GAA gene impacts normal exon 2 splicing and is found in two-thirds of late-onset Pompe...
Organism or Cell Type:
cell culture: fibroblast, forced-myogenic cells
Citation Extract:
Ham KA, Johnsen RD, Tchan M, Wilton SD, Aung-Htut MT. Developing Therapeutic Splice-Correcting Antisense Oligomers for Adult-Onset Pompe Disease with c.-32-13T>G Mutation. Methods Mol Biol. 2023;2587:239-251. doi: 10.1007/978-1-0716-2772-3_14.

Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy

Authors:
Stoodley J, Miraz DS, Jad Y, Fischer M, Wood MJA, Varela MA
Citation:
Methods Mol Biol. 2023;2587:209-237. doi: 10.1007/978-1-0716-2772-3_13
Epub:
Not Epub
Abstract:
Antisense oligonucleotides (ASOs) have shown great therapeutic potential in the treatment of many neuromuscular diseases...
Delivery Method:
peptide-linked
Organism or Cell Type:
mice
Citation Extract:
Stoodley J, Miraz DS, Jad Y, Fischer M, Wood MJA, Varela MA. Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy. Methods Mol Biol. 2023;2587:209-237. doi: 10.1007/978-1-0716-2772-3_13.

Morpholino-Mediated Exons 28-29 Skipping of Dysferlin and Characterization of Multiexon-skipped Dysferlin using RT-PCR

Authors:
Anwar S, Yokota T
Citation:
Immunoblotting, and Membrane Wounding Assay. Methods Mol Biol. 2023;2587:183-196. doi: 10.1007/978-1-0716-2772-3_11
Epub:
Not Epub
Abstract:
Dysferlinopathies are a group of disabling muscular dystrophies that includes limb girdle muscular dystrophy type 2B (LGMD2B),...
Citation Extract:
Anwar S, Yokota T. Morpholino-Mediated Exons 28-29 Skipping of Dysferlin and Characterization of Multiexon-skipped Dysferlin using RT-PCR. Immunoblotting, and Membrane Wounding Assay. Methods Mol Biol. 2023;2587:183-196. doi: 10.1007/978-1-0716-2772-3_11.

Restoring Dystrophin Expression with Exon 44 and 53 Skipping in the DMD Gene in Immortalized Myotubes

Authors:
Echigoya Y, Yokota T
Citation:
Methods Mol Biol. 2023;2587:125-139. doi: 10.1007/978-1-0716-2772-3_7
Epub:
Not Epub
Abstract:
Phosphorodiamidate morpholino oligomer (PMO)-mediated exon skipping is a therapeutic approach that applies to many Duchenne...
Organism or Cell Type:
cell culture: myotubes
Citation Extract:
Echigoya Y, Yokota T. Restoring Dystrophin Expression with Exon 44 and 53 Skipping in the DMD Gene in Immortalized Myotubes. Methods Mol Biol. 2023;2587:125-139. doi: 10.1007/978-1-0716-2772-3_7.

Restoring Dystrophin Expression by Skipping Exons 6 and 8 in Neonatal Dystrophic Dogs

Authors:
Shah MNA, Yokota T
Citation:
Methods Mol Biol. 2023;2587:107-124. doi: 10.1007/978-1-0716-2772-3_6
Epub:
Not Epub
Abstract:
Duchenne muscular dystrophy (DMD) is caused by the mutations in the DMD gene resulting in no dystrophin production. Skipping...
Organism or Cell Type:
Canis familiaris (dog) CXMDj
Citation Extract:
Shah MNA, Yokota T. Restoring Dystrophin Expression by Skipping Exons 6 and 8 in Neonatal Dystrophic Dogs. Methods Mol Biol. 2023;2587:107-124. doi: 10.1007/978-1-0716-2772-3_6.

Viltolarsen: From Preclinical Studies to FDA Approval

Authors:
Roshmi RR, Yokota T
Citation:
Methods Mol Biol. 2023;2587:31-41. doi: 10.1007/978-1-0716-2772-3_2
Epub:
Not Epub
Abstract:
Viltolarsen is a phosphorodiamidate morpholino antisense oligonucleotide (PMO) designed to skip exon 53 of the DMD gene for the...
Citation Extract:
Roshmi RR, Yokota T. Viltolarsen: From Preclinical Studies to FDA Approval. Methods Mol Biol. 2023;2587:31-41. doi: 10.1007/978-1-0716-2772-3_2.

DNAJB6 Isoform Specific Knockdown: Therapeutic Potential for LGMDD1

Authors:
Findlay AR, Paing MM, Daw JA, Bengoechea R, Pittman SK, Li S, Wang F, Miller TM, True HL, Weihl CC
Citation:
bioRxiv. 2022;[preprint] doi:10.1101/2022.11.17.516920
Epub:
Not Epub
Abstract:
Dominant missense mutations in DNAJB6, an HSP40 co-chaperone, cause limb girdle muscular dystrophy (LGMD) D1. No treatments are...
Delivery Method:
Vivo-Morpholino
Organism or Cell Type:
cell culture: mouse myotubes & human myotubes, mice
Citation Extract:
Findlay AR, Paing MM, Daw JA, Bengoechea R, Pittman SK, Li S, Wang F, Miller TM, True HL, Weihl CC. DNAJB6 Isoform Specific Knockdown: Therapeutic Potential for LGMDD1. bioRxiv. 2022;[preprint] doi:10.1101/2022.11.17.516920.

Functional identification of the zebrafish Interleukin-1 receptor in an embryonic model of Il-1β-induced systemic inflammation

Authors:
Sebo DJ, Fetsko AR, Phipps KK, Taylor MR
Citation:
Front Immunol. 2022 Oct 26;13:1039161. doi: 10.3389/fimmu.2022.1039161. eCollection 2022
Epub:
Not Epub
Abstract:
Interleukin-1β (IL-1β) is a potent proinflammatory cytokine that plays a vital role in the innate immune system. To observe the...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Sebo DJ, Fetsko AR, Phipps KK, Taylor MR. Functional identification of the zebrafish Interleukin-1 receptor in an embryonic model of Il-1β-induced systemic inflammation. Front Immunol. 2022 Oct 26;13:1039161. doi: 10.3389/fimmu.2022.1039161. eCollection 2022.

An ancestral Wnt-Brachyury feedback loop in axial patterning and recruitment of mesoderm-determining target genes

Authors:
Schwaiger M, Andrikou C, Dnyansagar R, Murguia PF, Paganos P, Voronov D, Zimmermann B, Lebedeva T, Schmidt HA, Genikhovich G, Benvenuto G, Arnone MI, Technau U
Citation:
Nat Ecol Evol. 2022;[Epub] doi:10.1038/s41559-022-01905-w
Epub:
Yes
Abstract:
Transcription factors are crucial drivers of cellular differentiation during animal development and often share ancient...
Citation Extract:
Schwaiger M, Andrikou C, Dnyansagar R, Murguia PF, Paganos P, Voronov D, Zimmermann B, Lebedeva T, Schmidt HA, Genikhovich G, Benvenuto G, Arnone MI, Technau U. An ancestral Wnt-Brachyury feedback loop in axial patterning and recruitment of mesoderm-determining target genes. Nat Ecol Evol. 2022;[Epub] doi:10.1038/s41559-022-01905-w.

Elf3 deficiency during zebrafish development alters extracellular matrix organization and disrupts tissue morphogenesis

Authors:
Sarmah S, Hawkins MR, Manikandan P, Farrell M, Marrs JA
Citation:
PLoS One. 2022 Nov 16;17(11):e0276255. doi: 10.1371/journal.pone.0276255. eCollection 2022
Epub:
Not Epub
Abstract:
E26 transformation specific (ETS) family transcription factors are expressed during embryogenesis and are involved in various...
Delivery Method:
microinjection
Organism or Cell Type:
zebrafish
Citation Extract:
Sarmah S, Hawkins MR, Manikandan P, Farrell M, Marrs JA. Elf3 deficiency during zebrafish development alters extracellular matrix organization and disrupts tissue morphogenesis. PLoS One. 2022 Nov 16;17(11):e0276255. doi: 10.1371/journal.pone.0276255. eCollection 2022.

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