AAV9 Gene Therapy in Type II GM1 Gangliosidosis — A Phase 1–2 Trial

Connor J. Lewis

Office of the Clinical Director, National Human Genome Research Institute, Bethesda, MD

Precilla D’Souza

Office of the Clinical Director, National Human Genome Research Institute, Bethesda, MD

Jean M. Johnston

Office of the Clinical Director, National Human Genome Research Institute, Bethesda, MD

Maria T. Acosta

Office of the Clinical Director, National Human Genome Research Institute, Bethesda, MD

Cristan Farmer

Neurodevelopmental and Behavioral Phenotyping Service, National Institute of Mental Health, Bethesda, MD

Eva H. Baker

Department of Diagnostic Radiology, National Institutes of Health Clinical Center, Bethesda, MD

Anna Crowell

Office of the Clinical Director, National Human Genome Research Institute, Bethesda, MD

Yoliann Mojica

Office of the Clinical Director, National Human Genome Research Institute, Bethesda, MD

Sumaiya Ashraf

Medical Genetics Branch, National Human Genome Research Institute, Bethesda, MD

Lisa Joseph

Neurodevelopmental and Behavioral Phenotyping Service, National Institute of Mental Health, Bethesda, MD

Gilbert Vézina

Division of Diagnostic Imaging and Radiology, Children’s National Hospital, Washington, DC

Zenaide Quezado

Department of Perioperative Medicine, National Institutes of Health Clinical Center, Bethesda, MD

Muhammad H. Yousef

Department of Perioperative Medicine, National Institutes of Health Clinical Center, Bethesda, MD

Zeynep Vardar

Department of Radiology, University of Massachusetts Chan Medical School, Worcester

Mohammed Salman Shazeeb

Department of Radiology, University of Massachusetts Chan Medical School, Worcester

Manuela Corti

Meghan Blackwood

Horae Gene Therapy Center and Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester

Kirsten Coleman

Powell Gene Therapy Center, University of Florida, Gainesville

Rita Batista

Horae Gene Therapy Center and Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester

Audrey Thurm

Neurodevelopmental and Behavioral Phenotyping Service, National Institute of Mental Health, Bethesda, MD

Erika De Boever

EDB Consulting, Haverford, PA

William A. Gahl

Medical Genetics Branch, National Human Genome Research Institute, Bethesda, MD

Barry J. Byrne

Department of Pediatrics, University of Florida, Gainesville

Terence R. Flotte

Horae Gene Therapy Center and Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester

Xuntian Jiang

Department of Medicine, Washington University School of Medicine, St. Louis

Amanda L. Gross

Scott-Ritchey Research Center, Department of Anatomy, Physiology, and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, AL

Allison M. Keeler

Horae Gene Therapy Center and Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester

Heather Gray-Edwards

Department of Radiology, University of Massachusetts Chan Medical School, Worcester

Douglas R. Martin

Scott-Ritchey Research Center, Department of Anatomy, Physiology, and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, AL

Miguel Sena-Esteves

Horae Gene Therapy Center and Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester

Cynthia J. Tifft

Office of the Clinical Director, National Human Genome Research Institute, Bethesda, MD