Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD

• Hlavní autoři: Britt Hanson (Autor), Sofia Stenler (Autor), Nina Ahlskog (Autor), Katarzyna Chwalenia (Autor), Nenad Svrzikapa (Autor), Anna M.L. Coenen-Stass (Autor), Marc S. Weinberg (Autor), Matthew J.A. Wood (Autor), Thomas C. Roberts (Autor)
• Médium: Kniha
• Vydáno: Elsevier, 2022-12-01T00:00:00Z.
• Témata: article
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Duchenne muscular dystrophy (DMD) is the most prevalent inherited myopathy affecting children, caused by genetic loss of the gene encoding the dystrophin protein. Here we have investigated the use of the Staphylococcus aureus CRISPR-Cas9 system and a double-cut strategy, delivered using a pair of ad...