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Article

Tunable Expression of an AAV Payload Using ADAR-mediated RNA Editing

2026-08-05

Abstract excerpt

A challenge of “once-and-done” adeno associated virus (AAV)-based gene therapy is the inability to modulate the level of therapeutic protein expression post-administration. Herein, we demonstrate the utility of an adenosine deaminase acting on RNA (ADAR) - mediated gene switch to control AAV-delivered gene expression. Using a premature termination codon (PTC) in the human Factor IX (hFIX) transgene, we established...

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Literature Corpus work
0c08f94a-6b07-5114-8ff0-b6033372c3fb
DOI
10.64898/2026.08.04.742838
Open publication

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Tunable Expression of an AAV Payload Using ADAR-mediated RNA EditingDOI 10.64898/2026.08.04.742838
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