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Phase 1/2 Clinical Trial of Combined Bilateral Intrathalamic/Intracisternal/Intrathecal Delivery of a rAAVrh8 vector in Infantile and Juvenile Tay-Sachs and Sandhoff Disease: Report of Ongoing Studies

T. R. Flotte,O. Cataltepe,H. L. Gray-Edwards, A. M. Keeler, R. Daci, B. Owusu-Adjei, R. Artinian, S. Parajuli, D. Kokoski, M. Blackwood,T. Taghian, M. S. Shazeeb, X. Jiang,H. Andonian, M. Kiefer, E. Townsend, A. Nagy,O. Rapalino, B. Wishart, R. H. Brown,M. Sena-Esteves,F. S. Eichler

HUMAN GENE THERAPY（2022）

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