A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing

A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing

Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by mutations in the X-linked dystrophin (DMD) gene. Exon deletions flanking exon 51, which disrupt the dystrophin open reading frame (ORF), represent one of the most common types of human DMD mutations. Previously, we used cl...

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Main Authors: Yu Zhang (Author), Hui Li (Author), Takahiko Nishiyama (Author), John R. McAnally (Author), Efrain Sanchez-Ortiz (Author), Jian Huang (Author), Pradeep P.A. Mammen (Author), Rhonda Bassel-Duby (Author), Eric N. Olson (Author)
Format: Book
Published: Elsevier, 2022-09-01T00:00:00Z.
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3rd Floor Main Library

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