Treating with Gamma-Secretase Modulators to Prevent Neurodegeneration in Mouse Models of Down Syndrome and Alzheimer Disease

Mobley, William C

Summary​

An extra copy of the gene for APP is necessary for Alzheimer disease (AD) in Down syndrome (DS); the APP products responsible include the 99 residue C-terminal fragment (C99) and Aβ42. Increased C99 and Aβ42 are present in mouse models of DS and of AD. To reduce C99 and Aβ42, and prevent or lessen neurodegeneration, we will treat with the γ-secretase modulator (GSM) BPN15606, a small molecule that increases γ-secretase activity and that lessened neurodegeneration in preliminary studies.

Down Syndrome Publications​