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

GSE292722 Homo sapiens Expression profiling by high throughput sequencing 6 samples Submitted 2025/06/01 Platform GPL34281
Summary
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.
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Common AAV gene therapy vectors show nonselective transduction of ex vivo human brain tissue
McGinnis JP, Ortiz-Guzman J, Guevara MC et al. · Molecular therapy. Methods & clinical development 2025 · PMID 40525125 · doi:10.1016/j.omtm.2025.101494
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Also filed as BioProject PRJNA1241144 and SRA study SRP572745. Searching any of these in the dataset finder brings you back here.

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