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Deep learning design and <i>in vivo</i> validation of Müller glia-specific <i>cis</i> -regulatory elements

2026-07-21

Abstract excerpt

Effective recombinant adeno-associated virus gene therapies require promoters that are compact and cell-type-specific. Ideally, promoters should also exhibit functional conservation when tested in model organisms to ensure that preclinical findings translate reliably to human patients. Here, we introduce a deep learning framework for designing cis -regulatory elements (CREs) meeting these criteria, applied to ret...

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Literature Corpus work
e239577e-c081-507a-b1cf-785e2f709538
DOI
10.64898/2026.07.15.738785
Open publication

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Deep learning design and <i>in vivo</i> validation of Müller glia-specific <i>cis</i> -regulatory elementsDOI 10.64898/2026.07.15.738785
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