Efficient exon skipping by base-editor-mediated abrogation of exonic splicing enhancers

Efficient exon skipping by base-editor-mediated abrogation of exonic splicing enhancers

Summary: Duchenne muscular dystrophy (DMD) is a severe genetic disease caused by the loss of the dystrophin protein. Exon skipping is a promising strategy to treat DMD by restoring truncated dystrophin. Here, we demonstrate that base editors (e.g., targeted AID-mediated mutagenesis [TAM]) are able t...

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Bibliographic Details
Main Authors: Han Qiu, Geng Li, Juanjuan Yuan, Dian Yang, Yunqing Ma, Feng Wang, Yi Dai, Xing Chang
Format: Article
Language:English
Published: Elsevier 2023-11-01
Series:Cell Reports
Subjects:
CP: Genomics
Online Access:http://www.sciencedirect.com/science/article/pii/S2211124723013529

Internet

http://www.sciencedirect.com/science/article/pii/S2211124723013529

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