Evaluating the Genomic Parameters Governing rAAV-Mediated Homologous Recombination.

ABSTRACT: Recombinant adeno-associated virus (rAAV) vectors have the unique ability to promote targeted integration of transgenes via homologous recombination at specified genomic sites, reaching frequencies of 0.1%-1%. We studied genomic parameters that influence targeting efficiencies on a large scale. To do this, we generated more than 1,000 engineered, doxycycline-inducible target sites in the human HAP1 cell line and infected this polyclonal population with a library of AAV-DJ targeting vectors, with each carrying a unique barcode. The heterogeneity of barcode integration at each target site provided an assessment of targeting efficiency at that locus. We compared targeting efficiency with and without target site transcription for identical chromosomal positions. Targeting efficiency was enhanc

SUBMITTER: Spector LP 

PROVIDER: S-EPMC7934627 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature