Cas9 targeting of toxic foci of RNA repeats
نویسندگان
چکیده
The sustained expression of RNA-targeting Cas9 delivered intramuscularly or systemically by adeno-associated viral vectors eliminates pathogenic foci expanded-repeat transcripts and reverses muscle-disease phenotypes in mouse models myotonic dystrophy type 1.
منابع مشابه
Cellular toxicity of expanded RNA repeats: focus on RNA foci
Discrete and punctate nuclear RNA foci are characteristic molecular hallmarks of pathogenesis in myotonic dystrophy type 1 and type 2. Intranuclear RNA inclusions of distinct morphology have also been found in fragile X-associated tremor ataxia syndrome, Huntington's disease-like 2, spinocerebellar ataxias type 8, type 10 and type 31. These neurological diseases are associated with the presence...
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ژورنال
عنوان ژورنال: Nature Biomedical Engineering
سال: 2021
ISSN: ['2157-846X']
DOI: https://doi.org/10.1038/s41551-021-00688-y