Project Grant R01NS138303

The National Institute of Neurological Disorders and Stroke, a component of the Department of Health and Human Services National Institutes of Health, awarded $614,689 to The General Hospital Corporation (doing business as Massachusetts General Hospital) on August 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to develop patient-based disease-modifying therapeutic strategies for Huntington's disease by targeting FAN1, an interstrand DNA-crosslink repair gene identified as a strong modifier of disease onset.

The research capitalizes on genome-wide association studies identifying FAN1 as the HD modifier gene producing the strongest onset modification signals in HD patients. Increased FAN1 levels due to a specific variant significantly delayed disease onset by decreasing CAG repeat instability, the primary driver of Huntington's disease. The project addresses critical questions bridging the gap between this patient-genetic target and effective disease-modifying treatments.

The award supports research performed at Massachusetts General Hospital's Research Management Division in Boston, Massachusetts, with a period of performance from August 1, 2026, through May 31, 2031. The assistance type is a Project Grant, classified as an R01 investigator-initiated research award. No sub-recipients are identified in the source materials.