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

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

Abstract 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 th...

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Bibliographic Details
Main Authors: Shani Attias Cohen, Orit Bar-Am, Claudia Fuoco, Galit Saar, Cesare Gargioli, Dror Seliktar
Format: Article
Language:English
Published: Nature Publishing Group 2022-09-01
Series:Cell Death and Disease
Online Access:https://doi.org/10.1038/s41419-022-05166-0

Internet

https://doi.org/10.1038/s41419-022-05166-0

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