Common AAV gene therapy vectors show nonselective transduction of ex vivo human brain tissue

ABSTRACT: We have adapted an ex vivo organotypic model to evaluate AAV transduction properties in living slices of human brain tissue. Using single-nucleus RNA sequencing, we found that common AAV vectors show broad transduction of normal cell types, with protein expression most apparent in astrocytes; this work introduces a pipeline for identifying and optimizing AAV gene therapy vectors in human brain samples.

ORGANISM(S): Homo sapiens

PROVIDER: GSE292722 | GEO | 2025/06/01

REPOSITORIES: GEO