Stem Cell Study
We have launched an initiative with the Stanford Institute for Stem Cell Biology and Regenerative Medicine and the Department of Neurosurgery at Stanford to develop patient-specific induced pluripotent cell lines (iPSCs) from patients with Parkinsonís disease with defined mutations and sporadic forms of the disease. Recent groundbreaking discoveries allow us now to use adult human skin cells, transduce them with specific genes, and generate cells that exhibit characteristics of embryonic stem cells, termed induced pluripotent stem cells (iPSCs). These lines will be used as an experimental pre-clinical model to study disease mechanisms unique to Parkinsonís disease. We predict that these cells will not only serve an Ďauthenticí model for Parkinsonís disease when further differentiated into the specific dopaminergic neurons, but that these cells are pathologically affected with Parkinsonís disease.
The objectives of this initiative are to (1) establish a bank of 15-20 iPSCs from patients with sporadic Parkinsonís disease and patients with defined mutations in genes associated with Parkinsonís disease, (2) differentiate iPSCs into dopaminergic neurons and assess neurochemical and neuropathological characteristics of Parkinsonís disease of these cells in vitro, and (3) test the hypothesis that specific pharmacologic agents can be used to block or reverse pathological characteristics.
We have generated the first iPCSs from patients with specific mutations in the SNCA gene and we are in the process of ascertaining selected patients for this study.
The absence of cellular models of Parkinsonís disease represents a major bottleneck in the scientific field of Parkinsonís disease, which, if solved, would be instantly translated into a wide range of clinical applications, including drug discovery. This is an essential avenue if we want to offer our patients a new therapeutic approach that can give them a near normal life after being diagnosed with this progressively disabling disease.