Promoter orientation within an AAV-CRISPR vector affects Cas9 expression and gene editing efficiency

Adeno-associated virus (AAV) vectors have been widely adopted for delivery of CRISPR-Cas components, especially for therapeutic gene editing. For a single vector system, both the Cas9 and guide RNA (gRNA) are encoded within a single transgene, usually from separate promoters. Careful design of this...

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Main Authors: Fry, LE, Peddle, CF, Stevanovic, M, Barnard, AR, McClements, ME, MacLaren, RE
格式: Journal article
语言:English
出版: Mary Ann Liebert Inc 2020