Comparative analysis of cell-specific promoters in AAV9-mediated gene therapy targeting the central nervous system.

ABSTRACT: We present a comprehensive toolkit of ubiquitous and cell-specific promoters for potential use in adeno-associated virus (AAV)9-mediated gene therapy for central nervous system (CNS) disorders, systematically evaluating biodistribution, cellular specificity, and peripheral targeting. While ubiquitous promoters, such as the cytomegalovirus early enhancer/chicken beta-actin (CAG) promoter, drive widespread expression, cell-specific promoters offer greater targeting precision. We introduce a novel, astrocyte-specific, truncated glial fibrillary acidic protein (GFAP) promoter, named gfaABCD1405 (gfa1405), which enhances astrocyte specificity while reducing size, improving utility for gene therapies requiring larger transgenes. gfa1405 demonstrated broader CNS expression than gfaABC(1)D, addres

SUBMITTER: Chornyy S 

PROVIDER: S-EPMC12481918 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature