Comparing molecular and computational approaches for detecting viral integration of AAV gene therapy constructs

ABSTRACT: Many current gene therapy targets use recombinant adeno-associated virus (AAV). The majority of delivered AAV therapeutics persist as episomes, separate from host DNA, yet some viral DNA can integrate into host DNA in different proportions and at genomic locations. The potential for viral integration leading to oncogenic transformation has led regulatory agencies to require investigation into AAV integration events following gene therapy in preclinical species. In the present study, tissues were collected from cynomolgus monkeys and mice 6 and 8 weeks, respectively, following administration of an AAV vector delivering transgene cargo. We compared three different next-generation sequencing approaches (shearing extension primer tag selection ligation-mediated PCR, targeted enrichment sequenc

SUBMITTER: Oziolor E 

PROVIDER: S-EPMC10209688 | biostudies-literature | 2023 May

REPOSITORIES: biostudies-literature