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CRISPR/Cas therapeutic strategies for autosomal dominant disorders

Autosomal dominant disorders present unique challenges, as therapeutics must often distinguish between healthy and diseased alleles while maintaining high efficiency, specificity, and safety. For this task, CRISPR/Cas remains particularly promising. Various CRISPR/Cas systems, like homology-directed...

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Detalles Bibliográficos
Autores principales:	Caruso, Salvatore Marco, Quinn, Peter M.J., da Costa, Bruna Lopes, Tsang, Stephen H.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society for Clinical Investigation 2022
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9057583/ https://www.ncbi.nlm.nih.gov/pubmed/35499084 http://dx.doi.org/10.1172/JCI158287

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