Single-swap editing for the correction of common Duchenne muscular dystrophy mutations

Single-swap editing for the correction of common Duchenne muscular dystrophy mutations

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive disease of progressive muscle weakness and wasting caused by the absence of dystrophin protein. Current gene therapy approaches using antisense oligonucleotides require lifelong dosing and have limited efficacy in restoring dystrophin p...

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Bibliographic Details
Main Authors: Andreas C. Chai, Francesco Chemello, Hui Li, Takahiko Nishiyama, Kenian Chen, Yu Zhang, Efraín Sánchez-Ortiz, Adeeb Alomar, Lin Xu, Ning Liu, Rhonda Bassel-Duby, Eric N. Olson
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA editing
base editing
CRISPR-Cas9
gene editing
exon skipping
DMD
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253123000951

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http://www.sciencedirect.com/science/article/pii/S2162253123000951

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