SCIEnTIFIC ADvISoRy BoARD

X. William Yang, MD, PhD, Vice Chair

David Geffen School of Medicine at UCLA

Anne B. Young, MD, PhD, Chair Emerita

Massachusetts General Hospital

Harvard Medical School

Richard I. Morimoto, PhD

Northwestern University

A. Jennifer Morton, PhD, ScD, FRSB

University of Cambridge

Richard C. Mulligan, PhD

Harvard Medical School

Harry T. Orr, PhD

University of Minnesota

Henry L. Paulson, MD, PhD

University of Michigan

Christopher E. Pearson, PhD

The Hospital for Sick Children (SickKids)

University of Toronto

Bernard M. Ravina, MD

Atlas Venture

Lynn A. Raymond, MD, PhD, FRCPC

University of British Columbia

H. Diana Rosas, MD

Massachusetts General Hospital

Harvard Medical School

Joan S. Steffan, PhD

University of California, Irvine

Sarah J. Tabrizi, FRCP, PhD, FMedSci

University College London (UCL) Institute of Neurology

Leslie P. Weiner, MD

University of Southern California

Nancy S. Wexler, PhD

Hereditary Disease Foundation

Columbia University

Ai Yamamoto, PhD

Columbia University

Andrew S. Yoo, PhD

Washington University School of Medicine

Scott Zeitlin, PhD

University of Virginia School of Medicine

ADvISoRy

LESLIE M. THoMPSon, PHD CHAIR, SCIEnTIFIC

Donald Bren and Chancellor’s Professor

Department of Psychiatry and Human Behavior

Department of Neurobiology and Behavior

University of California, Irvine

BoARD

Dr. Leslie Thompson has studied Huntington’s disease for most of her scientific career. She was a member of the the HDF’s Venezuela Project that identified the causative gene for HD in 1993. She is trying to understand how the HD mutation damages brain cells and identify targets for new drugs to prevent or ameliorate the damage. She is also looking at how the mutation influences modifications of the huntingtin protein and other cellular molecules. In addition, Dr. Thompson worked with a group of investigators to establish the HD patient-derived iPS cell consortium (induced pluripotent stem cells) and is using stem cells to study HD through multi-institutional collaborations and Big Data approaches. She is currently evaluating the use of human neural stem cells as a possible therapy for HD.

Dr. Thompson is an American Association for the Advancement of Science fellow. She received the HDF’s Leslie Gehry Brenner Prize for Innovation in Science in 2013.

DIAnE MERRy, PHD

vICE CHAIR, SCIEnTIFIC ADvISoRy BoARD

Professor and Vice Chair

Department of Biochemistry and Molecular Biology

Thomas Jefferson University

Dr. Diane Merry researches the molecular mechanisms underlying inherited neurodegenerative disease, with a primary focus on Kennedy’s disease, which is caused by the identical and unusual genetic mutation that causes Huntington’s disease. Dr. Merry’s group has developed and utilized unique cell and mouse models to both understand mechanisms of disease and identify molecular targets for therapeutic development. Over the past decades her group has made important and fundamental discoveries into the structural and functional requirements of the mutant androgen receptor (AR) protein in disease and has identified several AR targets that are in preclinical development.

Dr. Merry serves as Vice Chair of the Department of Biochemistry and Molecular Biology at the Sidney Kimmel Medical College of Thomas Jefferson University and chairs the Scientific Advisory Board for the Vickie & Jack Farber Institute for Neuroscience at Jefferson.

X. WILLIAM yAng, MD, PHD

vICE CHAIR, SCIEnTIFIC ADvISoRy BoARD

Professor

Terry Semel Chair in Alzheimer’s Disease Research and Treatment

Center for Neurobehavioral Genetics

The Jane and Terry Semel Institute for Neuroscience & Human Behavior

Department of Psychiatry & Biobehavioral Sciences, Brain Research Institute

David Geffen School of Medicine at UCLA

Dr. X. William Yang co-invented a powerful mouse genetic technology to engineer bacterial artificial chromosomes (BACs) and to generate BAC transgenic mice. His laboratory at UCLA has made significant contributions to the development of transgenic mouse models for human neurodegenerative disorders including Huntington’s, Parkinson’s and Alzheimer’s diseases, and the use of such models to dissect disease mechanisms and identify therapeutic targets.

The Yang lab has also applied novel genetic and systems biology approaches to study brain gene expression, and to decipher RNA and protein networks for HD in particular. They study the role of basal ganglia circuitry in the generation of normal and pathological behaviors. The Yang lab invented a new mouse genetic tool (called MORF mice) for brainwide labeling of thousands of neurons and glial cells to illuminate their exquisite morphology. Dr. Yang received the NIH’s BRAIN Initiative award, the McKnight Foundation’s Brain Disorder Award, and the HDF’s 2014 Leslie Gehry Brenner Prize for Innovation in Science. He is a member of the American Society for Clinical Investigation.

AnnE B. young, MD, PHD CHAIR EMERITA, SCIEnTIFIC ADvISoRy BoARD

Chair Emerita, Department of Neurology

Massachusetts General Hospital

Distinguished Julieanne Dorn Professor of Neurology, Harvard Medical School

Royal College of Physicians, London

American Academy of Arts and Sciences

National Academy of Medicine

Dr. Anne B. Young is an acclaimed researcher and clinician whose work at the bench and bedside has concentrated on neurotransmitter systems in the basal ganglia and their role in Huntington’s, Alzheimer’s and Parkinson’s diseases. She and her late husband John B. (“Jack”) Penney, Jr. first conceptualized a model of the functional anatomy of the basal ganglia that has been termed the “classical” model. They both played key roles in the HDF’s Venezuela Project which led to the identification of the HD gene.

Dr. Young was recruited in 1991 to Harvard Medical School and Massachusetts General Hospital as the hospital’s first female head of a department. She founded and designed the MassGeneral Institute for Neurodegenerative Diseases (MIND) in 2001 to accelerate the discovery of effective therapies for these diseases. In addition to training generations of outstanding clinicians and researchers, she has served on many editorial and advisory boards, served as president of the Society for Neuroscience, and received many awards and honors, including the HDF’s 2016 Leslie Gehry Brenner Prize for Innovation in Science.