Optimized genomic editing of a common Duchenne muscular dystrophy mutation in patient-derived muscle cells and a new humanized mouse model

Optimized genomic editing of a common Duchenne muscular dystrophy mutation in patient-derived muscle cells and a new humanized mouse model

Duchenne muscular dystrophy (DMD) is a fatal X-linked, recessive disease caused by mutations in the DMD gene encoding dystrophin, a membrane-associated protein necessary for maintaining muscle structure and function. One of the common DMD mutations is the deletion of exon 52 (Δ52), which introduces...

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Bibliographic Details
Main Authors: Mateusz Z. Durbacz, Yu Zhang, Hui Li, Takahiko Nishiyama, Efrain Sanchez- Ortiz, John R. McAnally, Damir Alzhanov, Ning Liu, Eric N. Olson
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA Editing
Duchenne muscular dystrophy
CRISPR
AAV
single-cut
gene editing
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253125001234
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