BioCentury
ARTICLE | Distillery Therapeutics

Neurology

June 13, 2018 6:17 PM UTC

Cell culture and fruit fly studies suggest promoting NAD+ synthesis could help treat PD caused by GBA mutations. In neurons generated via induced pluripotent stem (iPS) cells from a patient harboring a GBA mutation, levels of NAD+ and nicotinamide nucleotide adenylyltransferase 2 (NMNAT2), which participates in the biosynthesis of NAD+, were lower than in neurons derived from iPS cells engineered to express wild-type GBA. In a Drosophila model of GBA-mutant PD, the NAD+ precursor nicotinamide riboside decreased dopaminergic neuron loss and increased climbing ability compared with no treatment. Next steps could include testing the NAD+ precursor in additional animal models of PD...