Adenine base editing-mediated exon skipping restores dystrophin in humanized Duchenne mouse model
Abstract Duchenne muscular dystrophy (DMD) affecting 1 in 3500–5000 live male newborns is the frequently fatal genetic disease resulted from various mutations in DMD gene encoding dystrophin protein. About 70% of DMD-causing mutations are exon deletion leading to frameshift of open reading frame and...
Main Authors: | , , , , , , , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Nature Portfolio
2024-07-01
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Series: | Nature Communications |
Online Access: | https://doi.org/10.1038/s41467-024-50340-x |