Article

CRISPR-mediated correction of skeletal muscle Ca <sup>2+</sup> handling in a novel DMD patient-derived pluripotent stem cell model

2022-02-18

Abstract excerpt

Mutations in the dystrophin gene cause the most common and currently incurable Duchenne muscular dystrophy (DMD) characterized by progressive muscle wasting. Although abnormal Ca 2+ handling is a pathological feature of DMD, mechanisms underlying defective Ca 2+ homeostasis remain unclear. Here we generate a novel DMD patient-derived pluripotent stem cell (PSC) model of skeletal muscle with an isogenic control...

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Literature Corpus work: 29239323-7843-5a74-b4ce-6e7e0b442363
DOI: 10.1101/2022.02.17.480850

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