In vivo restoration of dystrophin expression in mdx mice using intra-muscular and intra-arterial injections of hydrogel microsphere carriers of exon skipping antisense oligonucleotides

Duchenne muscular dystrophy (DMD) is a genetic disease caused by a mutation in the X-linked Dytrophin gene preventing the expression of the functional protein. Exon skipping therapy using antisense oligonucleotides (AONs) is a promising therapeutic strategy for DMD. While benefits of AON therapy hav...

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Detalles Bibliográficos
Autores principales: Cohen, Shani Attias, Bar-Am, Orit, Fuoco, Claudia, Saar, Galit, Gargioli, Cesare, Seliktar, Dror
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9463190/
https://www.ncbi.nlm.nih.gov/pubmed/36085138
http://dx.doi.org/10.1038/s41419-022-05166-0

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