AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9
Abstract
Adeno-associated viral (AAV) vectors are a leading candidate for the delivery of CRISPR-Cas9 for therapeutic genome editing in vivo. However, AAV-based delivery involves persistent expression of the Cas9 nuclease, a bacterial protein. Recent studies indicate a high prevalence of neutralizing antibodies and T cells specific to the commonly used Cas9 orthologs from Streptococcus pyogenes (SpCas9) and Staphylococcus aureus (SaCas9) in humans. We...
Paper Details
Title
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9
Published Date
Jun 1, 2020
Journal
Volume
28
Issue
6
Pages
1432 - 1441
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