The Cell Function and Pathophysiology Core will be led by Dr. Ted Dawson and will function to determine disease-associated phenotypes from the differentiated PDiPS cell lines. In addition, the members of the Cell Function and Pathophysiology Core will be responsible for developing the existing phenotypic assays into a format amenable for large-scale drug screening. In conclusion, we think that the talented clinical investigators and translational scientists that comprise our current PDiPS Consortium are well placed to transition our groundbreaking work into a U24 format that meets the criteria outlined in the new RFA (RFA-NS-11-011, see Fig. 3 for Milestones).
|Sanders, Laurie H; Laganiere, Josee; Cooper, Oliver et al. (2014) LRRK2 mutations cause mitochondrial DNA damage in iPSC-derived neural cells from Parkinson's disease patients: reversal by gene correction. Neurobiol Dis 62:381-6|
|Sundberg, Maria; Bogetofte, Helle; Lawson, Tristan et al. (2013) Improved cell therapy protocols for Parkinson's disease based on differentiation efficiency and safety of hESC-, hiPSC-, and non-human primate iPSC-derived dopaminergic neurons. Stem Cells 31:1548-62|