Article

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

2022-10-01

Abstract excerpt

<h4>ABSTRACT</h4> 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 r...

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Literature Corpus work: cbe5fb89-3ad7-59cc-882a-2bd0955789a2
DOI: 10.1101/2022.09.30.510386

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