Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
<div>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 <em>Staphylococcus aureus</em> CRISPR-Cas9 system and a...
Главные авторы: | Hanson, B, Stenler, S, Ahlskog, N, Chwalenia, K, Svrzikapa, N, Coenen-Stass, AML, Weinberg, MS, Wood, MJA, Roberts, TC |
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Формат: | Journal article |
Язык: | English |
Опубликовано: |
Cell Press
2022
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