OSCR

Neuron-targeted gene therapy rescues multiple phenotypes of STXBP1-related disorders in mice and is well tolerated in nonhuman primates.

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Overview

Authors: Rangoli Aeran, Annie Tanenhaus, Sheila MS Sears, Nathan J Moerke, Adam Miller, Camille Artur, Yosr Bouhlal, Peter F Bove, Alexander J Diaz de Arce, Saki Shimizu, Jason Le, Keith Place, Dixon Hoffelt, Tselmeg Amarlkhagva, Jennifer Su, Ming Chen, Brooke A Babineau, John McLaughlin, Myat Soe, Warren Macdonald
and 13 other authorsI Winnie Lin, Dhruv Bole, Kristen M Valentine, Elizabeth Hallam, Puja Dhanota, Serena Liu, Steven A Tan, Ben Zhao, Raghavendra Hosur, Maria Candida Vila, Suresh Poda, Archana Belle, Stephanie Tagliatela
Published: 9 May 2025
Type: Research article
License: CC BY-NC-ND

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