Article
Generation and characterization of a mouse model of Becker muscular dystrophy with a deletion of Dmd exons 52 to 55.
Disease models & mechanisms - 1 Oct 2025
Perillat Lucie O M, Wong Tatianna W Y, Maino Eleonora, Ahmed Abdalla, Scott Ori, Hyatt Elzbieta, Delgado-Olguin Paul, Visuvanathan Shagana, Ivakine Evgueni A, Cohn Ronald D
Abstract excerpt
Becker muscular dystrophy (BMD) is a rare X-linked recessive neuromuscular disorder, frequently caused by in-frame deletions in the DMD gene that result in the production of a truncated, yet functional, dystrophin protein. The consequences of BMD-causing in-frame deletions on the organism are difficult to predict, especially in regard to long-term prognosis. Here, we used CRISPR-Cas9 to generate a new Dmd Δ52-55...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.