Original paper
A multifunctional AAV–CRISPR–Cas9 and its host response
Abstract
CRISPR-Cas9 delivery by adeno-associated virus (AAV) holds promise for gene therapy but faces critical barriers on account of its potential immunogenicity and limited payload capacity. Here, we demonstrate genome engineering in postnatal mice using AAV-split-Cas9, a multifunctional platform customizable for genome editing, transcriptional regulation, and other previously impracticable applications of AAV-CRISPR-Cas9. We identify crucial...
Paper Details
Title
A multifunctional AAV–CRISPR–Cas9 and its host response
Published Date
Sep 5, 2016
Journal
Volume
13
Issue
10
Pages
868 - 874
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Notes
History