Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors.

ABSTRACT: Adeno-associated viral (AAV) vectors have emerged as the preferred platform for in vivo gene transfer because of their combined efficacy and safety. However, insertional mutagenesis with the subsequent development of hepatocellular carcinomas (HCCs) has been recurrently noted in newborn mice treated with high doses of AAV, and more recently, the association of wild-type AAV integrations in a subset of human HCCs has been documented. Here, we address, in a comprehensive, prospective study, the long-term risk of tumorigenicity in young adult mice following delivery of single-stranded AAVs targeting liver. HCC incidence in mice treated with therapeutic and reporter AAVs was low, in contrast to what has been previously documented in mice treated as newborns with higher doses of AAV. Spe

SUBMITTER: Ferla R 

PROVIDER: S-EPMC7803627 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature