317. Screening S. Aureus CRISPR-Cas9 Paired-Guide RNAs for Efficient Targeted Deletion in Duchenne Muscular Dystrophy
Abstract
Duchenne muscular dystrophy (DMD) is a recessive X-linked neuromuscular disorder that results in progressive muscle degeneration and premature death. Most patients have exonic deletions in the dystrophin gene that result in a frameshift and nonfunctional protein. In contrast, Becker muscular dystrophy (BMD) patients carry a range of exonic deletions in dystrophin that do not disrupt the reading frame, leading to a much milder disease phenotype....
Paper Details
Title
317. Screening S. Aureus CRISPR-Cas9 Paired-Guide RNAs for Efficient Targeted Deletion in Duchenne Muscular Dystrophy
Published Date
May 1, 2016
Journal
Volume
24
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