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Adeno-associated Virus (AAV) Synthetic Inverted Terminal Repeat Enhances Tissue Specific Transduction and Altered Vector Induced Stress Response

Tomoko Hasegawa Naveen Vridhachalam Ethan Sud Nikolai Thanu Kalikiri Mark Ross Robert Toennisson Priscila Villanueva Alicia M. Chandler Liujiang Song J. Bower R. Samulski Matt Hirsch
Jul 2026 · bioRxiv · 0 citations
Biology

TL;DR

Data demonstrate that SynITRs elicit tissue-specific transduction enhancement and alter the cellular stress response and offer an alternative context to elucidate wtITR biology for targeted, enhanced, and potentially safer human gene therapy.

DOI Semantic Scholar
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