The goals of this study are to develop patient-specific cell lines from patients with Parkinson’s disease (PD) with defined mutations and sporadic forms of the disease. These lines will be used as an experimental pre-clinical model to study cellular phenotypes and disease mechanisms unique to PD. The specific objectives of these studies are to (1) demonstrate the feasibility to generate patient-specific cell lines from skin cells of patients, (2) show that these cells have the ability to differentiate into specific brain cells relevant for PD, (3) demonstrate intrinsic differences and biological processes of these cells compared to controls, and (4) show that these cells are capable to improve symptoms of PD in pre-clinical models.
Reprogramming of adult skin cells to a pluripotent state is the technique we are using in this proposal. Derivation of patient-specific pluripotent cell lines eliminates many challenges (e.g. source of tissue and cells, immune reaction and ethical considerations) and becomes an attractive avenue of research.
We have no cure for PD, or good long-term therapeutics without deleterious side-effects. Therefore, there is a great need for novel therapies to halt or reverse the disease, which could be cell replacement therapy.
Statement of Benefit to California:
This proposal provides real benefits and value to the state of California and its citizens in providing new approaches for stem cell therapy in Parkinson’s disease (PD). Reprogramming of adult skin cells to a pluripotent state is the underlying mechanism where this application is build on. Derivation of patient-specific pluripotent cell lines eliminates many challenges (e.g. source of tissue and cells, immune reaction and ethical considerations) and becomes an attractive avenue of research.
Approx. 36,000-60,000 people in the State of California are affected with this neurodegenerative disease that causes a high degree of disability and financial burden for our health care system. It is estimated that the number of PD cases will double by the year 2030. With this research project, we will evaluate patient-specific pluripotent cell lines in various pre-clinical conditions to get closer to therapeutic applications for PD, for which we have no cure for or adequate long-term therapies to date.