SCIEnTIFIC ADvISoRy BoARD

LESLIE P. WEInER, MD

Professor

Molecular Microbiology & Immunology, Neurology

Richard Angus Grant, Sr., Chair in Neurology

Keck School of Medicine

University of Southern California

Dr. Leslie Weiner is a world-renowned neurologist, educator and researcher. He is internationally recognized for his research on the T-cell vaccine for multiple sclerosis. Dr. Weiner served as chair of the USC Department of Neurology for 25 years, guiding its growth from 3 to 40 full-time faculty members. He was instrumental in building neuroscience at USC. The Leslie P. Wiener Neurological Care and Research Center is the hub of outpatient care on the USC Health Sciences campus. The Weiner Chair of Neurology was donated and named in his honor in 2002.

nAnCy S. WEXLER, PHD

President, Hereditary Disease Foundation

Higgins Professor of Neuropsychology, Columbia University

2019 Double Helix Medal

1993 Albert Lasker Public Service Award

Royal College of Physicians, London

American Academy of Arts and Sciences

National Academy of Medicine

Dr.Nancy Wexler has devoted her career to finding treatments and cures for Huntington’s disease. She led the HDF’s history making Venezuela Project, a 22- year international collaborative team, studying the world’s largest Huntington’s disease family in Venezuela, collecting tissue samples, and developing a family tree of over 18,000 individuals spanning 10 generations. This work led to the discovery of the DNA marker for Huntington’s disease in 1983 and the HD gene itself in 1993. This same genetic material has assisted in the mapping of other disease genes, including those responsible for familial Alzheimer’s disease, kidney cancer, two kinds of neurofibromatosis, Lou Gehrig’s disease (ALS), dwarfism and others. One important result of this work was the development of a genetic test to determine if an individual carries the expanded version of the HD gene.

In 2020, the HDF established the Nancy S. Wexler Young Investigator Prize to be awarded annually to a researcher whose work reflects the highest caliber of excellence, diligence and creative thinking. Dr. Wexler has received numerous awards and honors for her work including honorary doctorates from the University of Michigan, Bard College and Yale. She headed a landmark Congressional Commission on the Control and Consequences of Huntington’s Disease from 1976-1978 and chaired the Ethical, Legal and Social Implications Working Group of the Human Genome Project.

AI yAMAMoTo, PHD

Associate Professor of Neurology

Associate Professor of Pathology & Cell Biology

Columbia University

Dr. Ai Yamamoto’s interest in HD research started during college in the laboratory of Dr. Ann Graybiel at Massachusetts Institute of Technology when she learned how the striatum of the brain is central to coordinating movement. With HDF support, she performed her dissertation work with Dr. Rene Hen at Columbia University, where she and her colleague Jose Lucas used inducible mouse genetics to demonstrate that turning off disease-causing genes leads to the reversal of the HD-like symptoms. As a Helen Hay Whitney Postdoctoral Fellow in Dr. James E. Rothman’s laboratory at Yale, Dr. Yamamoto identified the importance of the pathway autophagy in clearing protein aggregates (clumps), a hallmark of HD. These studies have led to a long-standing collaboration with Dr. Anne Simonsen at the University of Oslo pursuing how the protein Alfy regulates the degradation of aggregates by autophagy and its potential impact on HD pathogenesis.

Dr. Yamamoto’s early recognition includes a Dean’s Award of Excellence for her dissertation work, and the Harold and Golden Lamport Award for Excellence in Clinical Science Research. She received the HDF’s Leslie Gehry Brenner Prize for Innovation in Science in 2020.

How can we harness the 21st century toolbox to bring technologies to test all our best ideas, to change the course of Huntington’s disease and to bring about new treatment options? With the concerted effort of scientists from around the world working on HD, we are much closer to reaching that goal.

X. William Yang, MD, PhD David Geffen School of Medicine at UCLA

AnDREW S. yoo, PHD Professor

Department of Developmental Biology

Washington University School of Medicine

Dr. Andrew Yoo has a long-standing interest in understanding genetic networks that govern cell fate choices during development. He obtained his PhD degree at Columbia University, where he studied Notch signaling target genes that regulate progenitor differentiation. As a postdoctoral fellow at Stanford University, Dr. Yoo made seminal discoveries of microRNAs as a molecular switch of chromatin remodeling complexes during neural development. He then leveraged these findings to pioneer the utility of microRNAs as cell reprogramming agents to turn human skin cells directly into neurons.

Since then, the Yoo lab has been developing microRNA-mediated neuronal conversion methods to generate disease-relevant neuronal subtypes from patients to model and study adult-onset neurodegenerative disorders, including Huntington’s disease, primary tauopathy, and Alzheimer’s disease. Dr. Yoo has received numerous awards and honors for his work, including the NIH Director’s New Innovator Award and the Presidential Early Career Award for Scientists and Engineers from the White House.