Children’s Nutrition Research Center Staff Publications

Language

English

Publication Date

6-12-2025

Journal

Molecular Therapy Methods & Clinical Development

DOI

10.1016/j.omtm.2025.101494

PMID

40525125

PMCID

PMC12169722

PubMedCentral® Posted Date

5-21-2025

PubMedCentral® Full Text Version

Post-print

Abstract

The ability to deliver a therapeutic sequence to a specific cell type in the human brain would make possible innumerable therapeutic options for some of our most challenging diseases; however, studies on adeno-associated virus (AAV) vector tropism have generally relied on animal models with limited translational utility. For this reason, establishing the tropism of common adeno-associated virus (AAV) vectors in living human brain tissue serves as an important baseline for further optimization, as well as a determination of human brain cell types transduced by clinically approved gene therapy vectors AAV2 and AAV9. We have adapted an

Keywords

gene therapy, human brain, AAV, AAV tropism, human brain organotypic slice culture

Published Open-Access

yes

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