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Research Spotlight Webinar: "Developing Transformative Tools: Gene Therapy for Huntington's Disease" with Beverly Davidson, Leslie Thompson and Jang-Ho Cha

  • Hereditary Disease Foundation 601 W168th Street, Suite 54 New York, NY, 10032 United States (map)

About the Speakers

Beverly L. Davidson, PhD
Children’s Hospital of Philadelphia
University of Pennsylvania
2023 Transformative Research Award, Principal Investigator

Beverly Davidson’s laboratory focuses on genetic diseases that affect the brain, investigating how mutant gene products contribute to disease, and why certain brain regions are more susceptible than others. Her team employs advanced molecular methods, sequencing and imaging in animal models, and a variety of molecular tools to test various hypotheses. Her lab is also developing next generation therapeutics for inherited disorders, including the engineering of novel gene therapy vector capsids and cargo to approach tissue and cell type specific treatments. Recent honors include the 2023 National Ataxia Foundation’s Dr. John W. Schut Research Achievement Award. She is immediate past president of the American Society of Gene and Cell Therapy, the largest international association of gene and cell therapy research. She is an elected member of both the American Academy of Arts Sciences and the National Academy of Medicine. Dr. Davidson received the HDF’s Leslie Gehry Brenner Prize for Innovation in Science in 2015.

Leslie M. Thompson, PhD
University of California, Irvine
2023 Transformative Research Award, Co-Investigator

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.

Jang-Ho J. Cha, MD, PhD
Latus Bio, Inc.
2023 Transformative Research Award, Co-Investigator

Jang-Cha is the Chief Scientific Officer, Latus Bio, Inc. Previously, he was the Global Head of Translational Medicine, Neuroscience at Novartis. He obtained his MD and PhD from the University of Michigan and did his residence in neurology at Massachusetts General Hospital in Boston, MA.  During his career at Massachusetts General Hospital, Dr. Cha held multiple positions culminating in Associate Professor of Neurology.

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