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Article

RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes

8 Apr 2023

Abstract excerpt

Adeno-associated virus (AAV)-based gene therapy could be facilitated by the development of molecular switches to control the magnitude and timing of expression of therapeutic transgenes. RNA interference (RNAi)-based approaches hold unique potential as a clinically proven modality to pharmacologically regulate AAV gene dosage in a sequence-specific manner. We present a generalizable RNAi-based rheostat wherein...

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PMID: 37031257
DOI: 10.1038/s41467-023-37774-5

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