Article

A Multispecies, Modality-Agnostic Scalable In Vivo Mosaic Screening Platform for Therapeutic Target Discovery

2026-02-28

Abstract excerpt

Validating therapeutic targets for complex diseases requires investigating gene functions within native tissue architectures rather than reductionist in vitro models. Here we present a modality-agnostic AAV-based in vivo high-throughput screening platform capable of delivering knockouts, gain-of-function, and synthetic miRNA knockdowns directly to cells within the diseased environment. This system scales to hundre...

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Literature Corpus work: 13bbec20-1641-5ac0-9586-bb269c0081ed
DOI: 10.64898/2026.02.26.708253

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