HIGHLIGHTS
- who: Megha Subramanian from the Alnylam Pharmaceuticals, Cambridge, MA, USA have published the article: RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes, in the Journal: (JOURNAL) of 11/Nov/2022
- what: For potential clinical development, the authors report potent and specific siRNA sequences that may allow selective regulation of transgenes while minimizing unintended off-target effects. The authors report a generalizable and clinically viable approach for dosage control of AAVdelivered cargos involving dynamic, robust, and reversible control via RNAi with benefit of infrequent dosing. The authors demonstrate reversal of RNAi-mediated transgene knockdown . . .
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