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Identification of a robust promoter in mouse and human hepatocytes by in vivo biopanning of a barcoded AAV library

21 Apr 2025

Abstract excerpt

Recombinant adeno-associated viruses (AAVs) are leading vectors for in vivo human gene therapy. An integral vector element is promoters, which control transgene expression in either a ubiquitous or cell-type-selective manner. Identifying optimal capsid-promoter combinations is challenging, especially when considering on- versus off-target expression. Here, we report a pipeline for in vivo promoter biopanning in...

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PMID: 40263935
DOI: 10.1016/j.ymthe.2025.04.027

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