Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is primarily caused by out-of-frame deletions in the dystrophin gene. Exon skipping using phosphorodiamidate morpholino oligomers (PMOs) converts out-of-frame to in-frame mutations, producing partially functional dystrophin. Four single-exon skipping PMOs are approv...

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Detalles Bibliográficos
Autores principales: Lim, Kenji Rowel Q., Woo, Stanley, Melo, Dyanna, Huang, Yiqing, Dzierlega, Kasia, Shah, Md Nur Ahad, Aslesh, Tejal, Roshmi, Rohini Roy, Echigoya, Yusuke, Maruyama, Rika, Moulton, Hong M., Yokota, Toshifumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: National Academy of Sciences 2022
Materias:
Biological Sciences
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8892351/
https://www.ncbi.nlm.nih.gov/pubmed/35193974
http://dx.doi.org/10.1073/pnas.2112546119

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