An episomal DNA vector platform for the persistent genetic modification of pluripotent stem cells and their differentiated progeny.

ABSTRACT: The genetic modification of stem cells (SCs) is typically achieved using integrating vectors, whose potential integrative genotoxicity and propensity for epigenetic silencing during differentiation limit their application. The genetic modification of cells should provide sustainable levels of transgene expression, without compromising the viability of a cell or its progeny. We developed nonviral, nonintegrating, and autonomously replicating minimally sized DNA nanovectors to persistently genetically modify SCs and their differentiated progeny without causing any molecular or genetic damage. These DNA vectors are capable of efficiently modifying murine and human pluripotent SCs with minimal impact and without differentiation-mediated transgene silencing or vector loss. We demonstrate that t

SUBMITTER: Roig-Merino A 

PROVIDER: S-EPMC8758943 | biostudies-literature | 2022 Jan

REPOSITORIES: biostudies-literature