A multi-functional AAV-CRISPR-Cas9 and its host response

ABSTRACT: CRISPR-Cas9 delivery by AAV holds promise for gene therapy but faces critical barriers due to its potential immunogenicity and limited payload capacity. Here, we demonstrate genome engineering in postnatal mice using AAV-split-Cas9, a multi-functional platform customizable for genome-editing, transcriptional regulation, and other previously impracticable AAV-CRISPR-Cas9 applications. We identify crucial parameters that impact efficacy and clinical translation of our platform, including viral biodistribution, editing efficiencies in various organs, antigenicity, immunological reactions, and physiological outcomes. These results reveal that AAV-CRISPR-Cas9 evokes host responses with distinct cellular and molecular signatures, but unlike alternative delivery methods, does not induce detectabl

ORGANISM(S): Mus musculus

SUBMITTER: Alex Ng 

PROVIDER: E-GEOD-84534 | biostudies-arrayexpress |

REPOSITORIES: biostudies-arrayexpress