N6-Furfuryladenine is protective in Huntington’s disease models by signaling huntingtin phosphorylation

Significance We have discovered a molecule derived from DNA-damage repair that can correct the lack of phosphorylation of mutant huntingtin, the cause of Huntington’s disease (HD). In a mouse model, treatment reverses HD-like disease, and we see the lowering of mutant huntingtin levels to normal. The mechanism of this molecule is that it is processed to make a signal for kinase activity essential for repairing DNA. This mechanism is critical when neurons are stressed and have very low or absent energy levels. We propose that this molecule is a type of signaling from DNA-damage repair that occurs at dangerously low ATP levels.

N6-Furfuryladenine is protective in Huntington’s disease models by signaling huntingtin phosphorylation Journal Articles