Genetic detective work by Brigham researchers offers insight into a novel therapeutic approach for treating Alzheimer’s disease
Alzheimer’s disease (AD) varies widely in its age of onset, presentation and severity. Recently, the SORL1 gene has received increased attention since variations in this gene have been associated with both early and late onset AD. However, little is known about how damage to SORL1 leads to disease. Using stem cells from patients with AD, investigators from Brigham and Women’s Hospital, a founding member of the Mass General Brigham healthcare system, found that loss of normal SORL1 function leads to a reduction in two key proteins known to be involved in AD and which play an essential role in the neurons of healthy individuals. Their results, published in Cell Reports, suggest a potential new strategy for AD treatment, especially for patients not responsive to existing therapies.
