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Direct Reprogramming of Human DMD Fibroblasts into Myotubes for In Vitro Evaluation of Antisense-Mediated Exon Skipping and Exons 45–55 Skipping Accompanied by Rescue of Dystrophin Expression

Antisense oligonucleotide-mediated exon skipping is a promising therapeutic approach for the treatment of various genetic diseases and a therapy which has gained significant traction in recent years following FDA approval of new antisense-based drugs. Exon skipping for Duchenne muscular dystrophy (D...

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Vydáno v:Methods Mol Biol
Hlavní autoři: Lee, Joshua J. A., Saito, Takashi, Duddy, William, Takeda, Shin’ichi, Yokota, Toshifumi
Médium: Artigo
Jazyk:Inglês
Vydáno: 2018
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6578579/
https://ncbi.nlm.nih.gov/pubmed/30171539
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/978-1-4939-8651-4_8
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