טוען...

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...

תיאור מלא

שמור ב:
מידע ביבליוגרפי
הוצא לאור ב:Methods Mol Biol
Main Authors: Lee, Joshua J. A., Saito, Takashi, Duddy, William, Takeda, Shin’ichi, Yokota, Toshifumi
פורמט: Artigo
שפה:Inglês
יצא לאור: 2018
נושאים:
גישה מקוונת: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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