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All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo

BACKGROUND: Clustered, regularly interspaced, short palindromic repeats (CRISPR) and CRISPR-associated proteins (Cas) have recently opened a new avenue for gene therapy. Cas9 nuclease guided by a single-guide RNA (sgRNA) has been extensively used for genome editing. Currently, three Cas9 orthologs h...

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Detalles Bibliográficos
Autores principales: Ibraheim, Raed, Song, Chun-Qing, Mir, Aamir, Amrani, Nadia, Xue, Wen, Sontheimer, Erik J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6146650/
https://www.ncbi.nlm.nih.gov/pubmed/30231914
http://dx.doi.org/10.1186/s13059-018-1515-0