The X-linked Becker muscular dystrophy (bmx) mouse models Becker muscular dystrophy via deletion of murine dystrophin exons 45-47.

Christopher R Heier,Nikki M McCormack,Christopher B Tully,James S Novak,Breanne L Newell-Stamper,Alan J Russell,Alyson A Fiorillo

Journal of cachexia, sarcopenia and muscle（2023）

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摘要

The bmx mouse recapitulates BMD disease phenotypes with histological, molecular and functional deficits. Importantly, it can inform both BMD pathology and DMD dystrophin restoration therapies. This novel model will enable further characterization of BMD disease progression, identification of biomarkers, identification of therapeutic targets and new preclinical drug studies aimed at developing therapies for BMD patients.

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关键词

Becker muscular dystrophy,Duchenne muscular dystrophy,dystrophin,dystrophin-associated proteins,exon skipping,inflammation,microRNAs

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