2025 Hereditary Disease Foundation Funded Researchers

Vanessa L. Casha, PhD
Mentor: Baljit Khakh, PhD
University of California, Los Angeles
Project Title: Uncovering new therapies for Huntington’s disease by examining subcompartments in brain cells

Francisco Garcia, PhD
Mentor: Myriam Heiman, PhD
Massachusetts Institute of Technology, Cambridge
Project Title: Viruses targeting blood vessels in the brain as a therapeutic strategy for Huntington’s disease

Shota Shibata, MD, PhD
Mentor: Ricardo Mouro Pinto, PhD
Massachusetts General Hospital, Harvard Medical School, Boston
Project Title: Developing a novel approach to monitoring Huntington’s disease progression

Takeshi Uenaka, MD, PhD
Mentor: Marius Wernig, PhD
Stanford University School of Medicine, CA
Project Title: Understanding how the brain’s immune cells can reduce huntingtin clumping within neurons in human stem cell models

Ranen Aviner, PhD  
Chan Zuckerberg Biohub, San Francisco, CA
Project Title: Developing gene therapies for Huntington’s disease that target the synthesis of mutant Huntingtin protein  

Amit Laxmikant Deshmukh, PhD
The Hospital for Sick Children (SickKids),Toronto, Ontario, Canada
Project Title: Disrupting FAN1 and MLH1 interaction to stop Huntington’s disease features 

Rachel J. Harding, PhD 
University of Toronto, Ontario, Canada
Project Title: Developing chemical tools to investigate HAP40, the partner of the Huntington’s disease protein

Michael Hayden, PhD, FRCP(C), FRSC with Chris Kay, PhD
University of British Columbia, Vancouver, Canada
Project Title: Investigating whether large CAG repeat expansions in the brain cause early onset of Huntington’s disease in patients with DNA sequence changes in the HD gene

Chris Kay, PhD with Michael Hayden, PhD, FRCP(C), FRSC 
University of British Columbia, Vancouver, Canada
Project Title: Investigating whether large CAG repeat expansions in the brain cause early onset of Huntington’s disease in patients with DNA sequence changes in the HD gene

Kimberly Kegel-Gleason, PhD
Massachusetts General Hospital, Harvard Medical School, Boston
Project Title: Fat metabolism in Huntington’s disease  

Seongwon Lee, PhD with Young Mi Oh, PhD
Mercer University, Macon, GA
Project Title: Exploring how TET1 helps protect the brain in Huntington’s disease

Christian Neri, PhD
Sorbonne University, Paris, France
Project Title: Extracellular vesicles as a source of new-generation biomarkers for Huntington’s disease

Christopher Ng, PhD
Massachusetts Institute of Technology, Cambridge
Project Title: Reversing the repeat expansion in Huntington’s disease with a DNA repair therapy

Young Mi Oh, PhD with Seongwon Lee, PhD 
Mercer University, Macon, GA 
Project Title: Exploring how TET1 helps protect the brain in Huntington’s disease

Christopher E. Pearson, PhD
The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada
Project Title: Reversing the Huntington’s disease mutation for treatment