Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model

Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model

Abstract Background Gene editing therapies in development for correcting out-of-frame DMD mutations in Duchenne muscular dystrophy aim to replicate benign spontaneous deletions. Deletion of 45–55 DMD exons (del45–55) was described in asymptomatic subjects, but recently serious skeletal and cardiac c...

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Bibliographic Details
Main Authors: Javier Poyatos-García, Patricia Soblechero-Martín, Alessandro Liquori, Andrea López-Martínez, Pilar Maestre, Elisa González-Romero, Rafael P. Vázquez-Manrique, Nuria Muelas, Gema García-García, Jessica Ohana, Virginia Arechavala-Gomeza, Juan J. Vílchez
Format: Article
Language:English
Published: BMC 2024-10-01
Series:Skeletal Muscle
Subjects:
Duchenne muscular dystrophy
Becker muscular dystrophy
CRISPR-Cas9
Gene therapy
Cell model
Dystrophin
Online Access:https://doi.org/10.1186/s13395-024-00353-3

Internet

https://doi.org/10.1186/s13395-024-00353-3

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