The Differentiation, Genetic Repair and Reporter Core will introduce essential new approaches to directed IPS cell differentiation, human genomic editing and cell purification for an emerging disease modeling technology. The Differentiation, Genetic Repair and Reporter Core is closely integrated with the Reprogramming CROs (Harvard Stem Cell Institute and New York Stem Cell Foundation) and the Cell Function and Pathophysiology Core to transition high quality patient-specific iPS cell lines into assays to examine the vulnerability of ventral midbrain (VM) dopaminergic (DA) neurons derived from patients with genetic forms of PD for translational research and drug discovery. The PDiPS Consortium plan is to use more resources and budget for this core in year 1 in order to accelerate the work to provide new protocols, training and genetically repaired iPS cells with reporters for the entire consortium. In year 2, the focus will be on collaboration and scientific needs for laboratories that may require differentiated cells into neurons prior to shipment to their laboratories.
|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|