CRISPR/Cas9 DNA cleavage at SNP-derived PAM enables both in vitro and in vivo KRT12 mutation-specific targeting

CRISPR/Cas9-based therapeutics hold the possibility for permanent treatment of genetic disease. The potency and specificity of this system has been used to target dominantly inherited conditions caused by heterozygous missense mutations through inclusion of the mutated base in the short-guide RNA (s...

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Detalles Bibliográficos
Autores principales: Courtney, D G, Moore, J E, Atkinson, S D, Maurizi, E, Allen, E H A, Pedrioli, D M L, McLean, W H I, Nesbit, M A, Moore, C B T
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Short Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4705418/
https://www.ncbi.nlm.nih.gov/pubmed/26289666
http://dx.doi.org/10.1038/gt.2015.82

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