The HDSA Scientific Advisory Board (SAB) is comprised of leading experts in their fields. The Scientific Advisory Board's role is to advise the Board of Trustees and HDSA Management on a range of issues. In general, the SAB provides scientific review of research proposals to ensure that the research programs at HDSA are scientifically sound, pertinent and provide a high impact to the HD research community.
The SAB can be called upon to advise HDSA on any scientific issues that may arise (e.g., stem cell policy, use of animals in research).
The Committee’s specific responsibilities include:
Family Representative Novartis, Marketing Director
University of California at Irvine Professor, Psychiatry & Human Behavior
Senior Scientist, CMMT and BCCHRI UBC Director, Transgenic Core Facility Professor, Department of Medical Genetics UBC Neurogenetics Director of Research & Neurologist, Centre for Huntington Disease
Teva, Director of CNS Discovery
CHDI Foundation, Director of Model Systems
Novartis, Global Head of Translational Medicine Neuroscience
Dr. Cha is the Global Head Translational Medicine, Neuroscience at Novartis Institute for BioMedical Research. Dr. Cha obtained his M.D and Ph.D. from the University of Michigan and did his residency in Neurology at Massachusetts General Hospital in Boston, MA. During his career at Massachusetts General Hospital at the New England HDSA Center of Excellence, Dr. Cha held multiple positions culminating in Associate Professor of Neurology.
Active in the HD community, he has held several leadership positions, such as Chair of HDSA’s National Board of Trustees, Chair of the Gordon Research Conference on ‘CAG Triplet Repeat Disorders’ and chair of the Huntington Study Group HD Clinical Research Symposium. Internationally he has worked with the US-Venezuela Collaborative Huntington’s Disease Project for Hereditary Disease Foundation and with the Huntington Study Group. Dr. Cha is a regular speaker for the Huntington’s Disease Society of America and has presented regularly at the Annual HDSA Convention, local Chapter educational events and support groups and on a Caregiver’s Corner webinar.
Scientific Advisory Board Chair
Dr. Michelle Gray is currently an Assistant Professor in the Department of Neurology at the University of Alabama at Birmingham. She is the Dixon Scholar in Neuroscience, focused on the study of Huntington’s Disease. Dr. Gray earned her Ph.D. from the Ohio State University and performed post-doctoral training at the University of California, Los Angeles.
Dr. Gray is a laboratory scientist who has worked on Huntington’s disease (HD) for the last 10 years. She has been an invited speaker and presenter of her work at multiple Huntington’s Disease focused meetings including those sponsored by the Hereditary Disease Foundation and CHDI,Inc. In addition, she presents updates on Huntington’s disease to the Alabama Academy of Neurology and participates in Huntington’s Disease Education Day sponsored by the HDSA Center for Excellence at the University of Alabama at Birmingham.
Currently her work focuses on understanding the overall contribution of glial cells, which constitute about 50% of the cells in the brain, to Huntington’s disease. This includes understanding the normal function of the huntingtin protein in glial cells and determining how the mutant protein in glial cells contributes to neuronal dysfunction in HD.
Professor of Medicine and Cell Biology Chief, Endocrinology and Metabolism Co-Director, Neuro-Therapeutics Institute University of Massachusetts Medical School
Dr. Aronin’s research focuses on developing gene silencing for therapy in Huntington’s disease, an autosomal dominant neurodegenerative disease. The laboratory uses allele selective RNAi to silence the mutant huntingtin and preserve wild type huntingtin. The research team explores potential therapies and neuronal pathophysiology attributable to the mutant huntingtin. The team also studies viral delivery of allele selective shRNAmir, with single stranded and self-complementary adeno-associated virus. Dr. Aronin’s team studies next-generation therapeutics that will be applicable to a spectrum of autosomal dominant diseases, as well as studying mechanisms of pathogenesis in Huntington’s disease: mutant huntingtin mRNA kinetics, vesicle recycling and roles of proteins that interact with mutant huntingtin.
Assistant Professor, Burnett School of Biomedical Sciences, Division of Neuroscience
Dr. Amber L. Southwell, Assistant Professor, Burnett School of Biomedical Sciences, Division of Neuroscience, has been working in preclinical therapy development for Huntington disease (HD) since 2002. In 2009 she earned her PhD at the California Institute of Technology working with Dr. Paul Patterson to develop an intrabody gene therapy for HD. From 2009-2016, she did postdoctoral research at the University of British Columbia with Dr. Michael Hayden where she developed several novel mouse models of HD, a selective mutant huntingtin gene silencing therapy, and a CSF biomarker for brain huntingtin. She began her laboratory research group at the University of Central Florida in January 2017, where she continues these studies as well as new research on modifiers of disease onset.
NIH Distinguished Investigator Chief, Neurogenetics Branch
Dr. Fischbeck received A.B. and A.M. degrees from Harvard University and an M.D. degree from Johns Hopkins. After a medical internship at Case Western Reserve University and a neurology residency at the University of California in San Francisco, he did postdoctoral research on muscular dystrophy at the University of Pennsylvania. In 1982 he joined the faculty in the Neurology Department at the University of Pennsylvania Medical School. In 1998 he came to the NINDS as Chief of the Neurogenetics Branch. He received the Cotzias Award from the American Academy of Neurology and the Jacoby Award from the American Neurological Association, and he was elected to the Institute of Medicine. His research group is identifying the causes and studying the mechanisms of hereditary neurological and neuromuscular diseases with the goal of developing effective treatment for these disorders.
Professor of Neurology Harvard Medical School and the Massachusetts General Hospital Boston, MA
Dr. Marcy MacDonald obtained her Ph.D. in Medical Biophysics from the University of Toronto in 1980 and is currently a Professor of Neurology (Genetics) at Harvard Medical School and the Massachusetts General Hospital, where she is a founding member of the Center for Human Genetic Research.
Dr. MacDonald became involved in Huntington’s disease research in 1985, joining Dr. James Gusella in an international collaboration that identified the Huntington’s disease gene in 1993, improving the diagnosis and management of the disorder. Her research pioneers the use of genetic mouse models and genetic studies in human samples to contribute knowledge essential to the development of effective therapies for Huntington’s disease.
Dr. MacDonald has served the HDSA as co-chair of the Grants and Fellowship Committee (1998-2003). She is the Chair of the Medical and Scientific Advisory Committee (2008-2011) and for the more than two decades has spoken about Huntington’s disease research at HDSA events for local chapters and at the annual convention.
Tulloch Professor of Genetics, Department of Laboratory Medicine and Pathology Director, Institute of Translational Neuroscience University of Minnesota
Harry Orr received his Ph.D. from Washington University and completed his postdoctoral fellowship at Harvard. Dr. Orr’s research explores genes that play a role in neuron deterioration. This offers important implications for developing gene therapy and other types of treatments for patients with neurodegenerative diseases. Orr discovered the genetic basis for spinocerebellar ataxia type 1, an inherited and ultimately fatal movement disorder, like HD SCA1 is due to a polyglutamine expansion. Orr directs the Institute of Translational Neuroscience, created to translate laboratory research discoveries into clinical trials of new therapies.