Towards personalised allele-specific CRISPR gene editing to treat autosomal dominant disorders

CRISPR/Cas9 holds immense potential to treat a range of genetic disorders. Allele-specific gene disruption induced by non-homologous end-joining (NHEJ) DNA repair offers a potential treatment option for autosomal dominant disease. Here, we successfully delivered a plasmid encoding S. pyogenes Cas9 a...

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Detalhes bibliográficos
Publicado no:Sci Rep
Main Authors: Christie, Kathleen A., Courtney, David G., DeDionisio, Larry A., Shern, Connie Chao, De Majumdar, Shyamasree, Mairs, Laura C., Nesbit, M. Andrew, Moore, C. B. Tara
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2017
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5701044/
https://ncbi.nlm.nih.gov/pubmed/29170458
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-017-16279-4
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