Identification of adeno-associated virus variants for gene transfer into human neural cell types by parallel capsid screening.

ABSTRACT: Human brain cells generated by in vitro cell programming provide exciting prospects for disease modeling, drug discovery and cell therapy. These applications frequently require efficient and clinically compliant tools for genetic modification of the cells. Recombinant adeno-associated viruses (AAVs) fulfill these prerequisites for a number of reasons, including the availability of a myriad of AAV capsid variants with distinct cell type specificity (also called tropism). Here, we harnessed a customizable parallel screening approach to assess a panel of natural or synthetic AAV capsid variants for their efficacy in lineage-related human neural cell types. We identified common lead candidates suited for the transduction of directly converted, early-stage induced neural stem cells (iNSCs), ind

SUBMITTER: Flitsch LJ 

PROVIDER: S-EPMC9120183 | biostudies-literature | 2022 May

REPOSITORIES: biostudies-literature