Treating Muscular Dystrophy with CRISPR Gene Editing
Type
Blog Post
Published
Jan. 26, 2016, 3:30 p.m.
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Duchenne muscular dystrophy: The most common inherited disease
The genetic...genetic disease targeted is Duchenne muscular dystrophy (DMD), an X-linked recessive disorder affecting approximately...symptoms, a condition known as Becker muscular dystrophy (BMD).
Dystrophin’s size makes it difficult to...Exon skipping strategies for Duchenne muscular dystrophy. gRNAs can be designed to flank a single or multiple...Kaiser J. CRISPR helps heal mice with muscular dystrophy. Science. 2015 Dec 31. doi:10.1126/science.aae0169...dystrophin expression in a mouse model of muscular dystrophy. Science. 2015 Dec 31. pii: aad5725. PubMed PMID...function in a mouse model of Duchenne muscular dystrophy. Science. 2015 Dec 31. pii: aad5143. PubMed PMID...