Article

Common AAV gene therapy vectors show indiscriminate transduction of living human brain cell types

2024-11-15

Abstract excerpt

The development of cell-type-specific gene therapy vectors for treating neurological diseases holds great promise, but has relied on animal models with limited translational utility. We have adapted an ex vivo organotypic model to evaluate adeno-associated virus (AAV) transduction properties in living slices of human brain tissue. Using fluorescent reporter expression and single-nucleus RNA sequencing, we found t...

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Identifiers and source

Literature Corpus work: 90cd001e-513d-5345-82c3-8e4983dd8cb3
DOI: 10.1101/2024.11.14.623624

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