Federal Grant Award Summary

The University of Illinois received a $225,474 Project Grant from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853), awarded June 1, 2026, with a completion date of May 31, 2027. The grant funds the development of a base editing gene therapy to treat Parkinson's Disease by modulating the LRRK2 gene. The research delivers two primary therapeutic strategies: (1) evaluation of LRRK2 knockout using base editors to introduce premature stop codons, and (2) assessment of base editing to create LRRK2 variants with reduced activity. The project employs intein-split base editors delivered via adeno-associated viruses (AAVs) and will be tested in disease-relevant cellular and animal models of Parkinson's Disease.

This research addresses a significant therapeutic gap, as current Parkinson's Disease treatments are limited to symptomatic management despite mutations in the LRRK2 gene being major contributors to both familial and sporadic forms of the disease. The base editing approach offers advantages over existing therapeutic strategies by enabling permanent genetic modifications following single administration without generating double-strand DNA breaks, while also addressing key safety and efficacy questions related to genome editing in the central nervous system. Research activities will be conducted at the University of Illinois in Urbana, Illinois.