Federal Project Grant Award Summary

This $132,837 project grant, awarded on May 1, 2026, by the National Eye Institute under the Vision Research program (CFDA 93.867), funds research investigating SARM1 as a therapeutic target for two hereditary optic neuropathies: Autosomal Dominant Optic Atrophy (ADOA) and Leber Hereditary Optic Neuropathy (LHON). The research, conducted at Boston Children's Hospital in Massachusetts through April 30, 2028, aims to develop a novel therapeutic approach targeting mitochondria-induced neurodegeneration in retinal ganglion cells. The project builds upon preliminary findings demonstrating that SARM1 knockout nearly completely protects against degenerative phenotypes in a novel ADOA mouse model carrying the pathogenic OPA1R290Q/+ mutation, suggesting that inhibiting SARM1 represents a promising treatment strategy for both conditions.

The project will generate research products including mechanistic insights into the mitochondria-SARM1 pathway contributing to retinal ganglion cell death, empirical validation of SARM1 as a therapeutic target across two distinct hereditary optic neuropathies, and preclinical development of SARM1-targeted therapeutic approaches. These deliverables address a critical unmet medical need, as no effective treatments currently exist for ADOA or LHON, conditions that cause progressive vision loss through mitochondrial dysfunction.