Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice

Current therapies for Duchenne muscular dystrophy (DMD) use phosphorodiamidate morpholino oligomers (PMO) to induce exon skipping in the dystrophin pre-mRNA, enabling the translation of a shortened but functional dystrophin protein. This strategy has been hampered by insufficient delivery of PMO to...

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Detalles Bibliográficos
Autores principales: Desjardins, Cody A, Yao, Monica, Hall, John, O’Donnell, Emma, Venkatesan, Reshmii, Spring, Sean, Wen, Aiyun, Hsia, Nelson, Shen, Peiyi, Russo, Ryan, Lan, Bo, Picariello, Tyler, Tang, Kim, Weeden, Timothy, Zanotti, Stefano, Subramanian, Romesh, Ibraghimov-Beskrovnaya, Oxana
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
NAR Breakthrough Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9723632/
https://www.ncbi.nlm.nih.gov/pubmed/35944903
http://dx.doi.org/10.1093/nar/gkac641

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