病毒载体遗传毒性评价方法研究进展OA北大核心CSTPCD

With the gradual entry of gene therapy products into the market,there is an urgent need to establish viral vector-mediated genotoxicity evaluation methods.The two most widely used viral vectors are lentiviral vectors and adeno-associated viral vectors.Lentiviral vectors can stably integrate into the host genome while the viral vector genome mainly exists in the form of episomes in the nucleus of the host cell,and can still be randomly integrated into the host cell genome at a low frequency,the integra-tion modes for which include random integration and targeted integration.This paper reviews the existing methods for evaluating the genotoxicity mediated by viral vectors,including whole-genome analysis of off-target modifications,integration site analysis,karyotype analysis,and tests to assess the potential for cell transformation.This article also offers a vision of the establishment of an accurate and rapid evaluation system for viral vector-mediated genotoxicity in the hopes of providing reference for improvement and development of new methods for evaluating the genotoxicity of viral vectors.