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Phenotypic Rescue of STXBP1 in Vitro and in Vivo Models Using AAV-STXBP1 Gene Replacement Approach

Sijia Wu, Brian Harvey, Elena Morozova, Ayan Ghoshal, Adam Sheehy, Kevin Ghaemi, Moore Arnold, Manzhao Long, Chris Hinckley, Sharanya Unnikrishnan, Nicholas B. Gallo, Anh Nhu,Ramiro Massol, Chris Ehrenfels,Pete Clarner,Jinkuk Choi,Davide Gianni, Wan-Hung Lee, Kelly Bales

MOLECULAR THERAPY(2024)

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