Mutation-Independent Allele-Specific Editing by CRISPR-Cas9, a Novel Approach to Treat Autosomal Dominant Disease

CRISPR-Cas9 provides a tool to treat autosomal dominant disease by non-homologous end joining (NHEJ) gene disruption of the mutant allele. In order to discriminate between wild-type and mutant alleles, Streptococcus pyogenes Cas9 (SpCas9) must be able to detect a single nucleotide change. Allele-spe...

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Publicat a:Mol Ther
Autors principals: Christie, Kathleen A., Robertson, Louise J., Conway, Caroline, Blighe, Kevin, DeDionisio, Larry A., Chao-Shern, Connie, Kowalczyk, Amanda M., Marshall, John, Turnbull, Doug, Nesbit, M. Andrew, Moore, C.B. Tara
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2020
Matèries:
Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7403340/
https://ncbi.nlm.nih.gov/pubmed/32416058
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.05.002
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