Correction of DMD in human iPSC-derived cardiomyocytes by base-editing-induced exon skipping

Correction of DMD in human iPSC-derived cardiomyocytes by base-editing-induced exon skipping

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene. Previously, we showed that adenine base editing (ABE) can efficiently correct a nonsense point mutation in a DMD mouse model. Here, we explored the feasibility of base-editing-mediated exon skipping as a therapeutic strategy f...

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Bibliographic Details
Main Authors: Peipei Wang, Haiwen Li, Mandi Zhu, Rena Y. Han, Shuliang Guo, Renzhi Han
Format: Article
Language:English
Published: Elsevier 2023-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
ABE
adenine base editor
base editing
cardiomyocyte
cardiomyopathy
DMD
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122001693

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http://www.sciencedirect.com/science/article/pii/S2329050122001693

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