Decades of research have established two critical aspects of neurodegenerative diseases like Parkinson’s and Alzheimer’s: first, they are caused by the clumping or misfolding of certain proteins in specific brain cells; second, there are a growing number of genes that predispose patients to these diseases.
In two recent Cell Systems studies, a team of investigators co-led by HSCI Principal Faculty member Vikram Khurana have established new methods to connect the dots between the genetic factors and the misfolding proteins. The researchers mapped out how the clumping of the rogue protein in Parkinson’s disease, alpha-synuclein, is related to multiple genetic risk factors for the disease. Using these maps, they were able to identify pathologies in neurons derived from Parkinson’s patients with different genetic forms of the disease.
“This research brings us closer to a day when we can map out the exact cause of Parkinson’s in a specific patient, develop a personalized model from that patient’s stem cells, and target a treatment to the patient,” says Khurana.