Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors

Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors

Duchenne muscular dystrophy is an X-linked monogenic disease caused by mutations in the dystrophin gene (DMD) characterized by progressive muscle weakness, leading to loss of ambulation and decreased life expectancy. Since the current standard of care for Duchenne muscular dystrophy is to merely tre...

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Bibliographic Details
Main Authors: Michael Gapinske, Jackson Winter, Devyani Swami, Lauren Gapinske, Wendy S. Woods, Shraddha Shirguppe, Angelo Miskalis, Anna Busza, Dana Joulani, Collin J. Kao, Kurt Kostan, Anne Bigot, Rashid Bashir, Pablo Perez-Pinera
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA editing
gene editing
CRISPR-Cas9
Duchenne muscular dystrophy
exon skipping
adenine base editing
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253123002020

Internet

http://www.sciencedirect.com/science/article/pii/S2162253123002020

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