Gene mutation

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Editing out muscular dystrophy with crispr independent of gene mutation Duchenne Muscular Dystrophy, Medical Oncology, Muscular Dystrophy, Gene Therapy, Fda Approved, Disease, Human

Congenital muscular dystrophy type 1A (MDC1A) is caused by mutations in the Lama2 gene that lead to muscle wasting and the destruction of the protective myelin coating around peripheral nerves. | A Canadian team has developed a CRISPR-based gene activation technique that treats one type of congenital muscular dystrophy type regardless of the mutation type. The system has shown promise in mouse models of the disease.

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