Project Grant R01NS141924

The National Institute of Neurological Disorders and Stroke awarded President and Fellows of Harvard College $595,961 on September 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to investigate mitochondrial DNA mutations and gene editing approaches in neural dysfunction.

The research addresses mechanisms of central nervous system dysfunction triggered by mitochondrial DNA (mtDNA) mutations, particularly mutations in mitochondrial transfer RNA genes associated with neurological conditions including MELAS syndrome. The project leverages CRISPR/Cas9 gene editing technology to overcome limitations of prior mitochondrial-targeted approaches, enabling simplified target sequence design, multiple editing sites, and more flexible targeting options compared to transcription activator-like effector nuclease (TALEN) strategies. This approach is designed to clarify causality between specific mtDNA variants and neurological pathology by systematically testing mtDNA editing in cell models expressing suspected causal mutations.

The award supports research performed at Harvard's Cambridge, Massachusetts campus through the President and Fellows of Harvard College's Office for Sponsored Programs. The period of performance runs from September 1, 2026 through June 30, 2031. The assistance type is a Project Grant, which provides support for single or discrete, circumscribed projects of specified duration.