A phase 1/2 clinical trial of systemic gene transfer of scAAV9.U1a.HSGSH for MPS IIIA: Safety,tolerability, and preliminary evidence of biopotency期刊界 All Journals 搜尽天下杂志传播学术成果专业期刊搜索期刊信息化学术搜索

A phase 1/2 clinical trial of systemic gene transfer of scAAV9.U1a.HSGSH for MPS IIIA: Safety,tolerability, and preliminary evidence of biopotency

Authors:	K.M. Flanigan K.V. Truxal K.L. McBride K.A. McNally K.L. Kunkler N.A. Zumberge L. Martin S. Aylward M. Corridore Juan Ruiz C. McKee D. McCarty T.R. Simmons

Affiliation:	1. Center for Gene Therapy, Nationwide Children''s Hospital, Columbus, OH, United States;2. Center for Cardiovascular and Pulmonary Research, Nationwide Children''s Hospital, Columbus, OH, United States;3. Center for Biobehavioral Health, Nationwide Children''s Hospital, Columbus, OH, United States;4. Department of Radiology, Nationwide Children''s Hospital, Columbus, OH, United States;5. Division of Neurology, Nationwide Children''s Hospital, Columbus, OH, United States;6. Abeona Therapeutics Inc., Cleveland, OH, United States;g. Department of Anesthesia, Nationwide Children''s Hospital, Columbus, OH, United States

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