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Robust Huntingtin knockdown in cortex and putamen in large mammals using a novel dosing paradigm with VY-HTT01, an AAV gene therapy targeting Huntington for the treatment of Huntington's disease

P. Zhou, J. Carroll, F. Chen, J. Thompson,J. Aubin,E. Christensen,L. Liu,T. Nguyen,M. Scheel,P. Pechan,A. Kells, C. Gamba-Vitalo,S. Hersch,J. Hou,T. Carter,D. Sah

HUMAN GENE THERAPY(2018)

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