… for introducing functional versions of these mutated genes into cells that are affected by PD could provide an … to determine a more efficient way to introduce genes into the parts of the brain affected in PD. Hypothesis:  We … that may be useful for treating or curing PD. Impact on Diagnosis/Treatment of Parkinson’s disease:  This project …

… methodologies, these ZFN reagents will be injected into early stage pre-clinical model embryos, causing a single … are considered “knockouts” for the gene. Relevance to Diagnosis/Treatment of Parkinson’s Disease: Pre-clinical … MJFF Research Grant …

… This grant builds upon the research from a prior grant: Targeting the Lysosomal Pathway … GCase mutations. We then converted these stem cells into dopamine-producing nerve cells. We will use these to … using a sophisticated, powerful microscope. Impact on Diagnosis/Treatment of Parkinson's Disease: Several …

… in Science that administration of synthetic alpha-synuclein into pre-formed fibrils in pre-clinical models was … disease (PD), providing a valuable tool for pre-clinical research. Objectives for Supplemental Investigation: The … data will be presented and discussed. Importance of This Research for the Development of a New PD Therapy: In the …

… revealed a role for PINK1 and PARKIN , two genes mutated in early onset Parkinson’s disease, function to promote a … PARKIN pathways and then extended to tissues. Impact on Diagnosis/Treatment of Parkinson’s Disease:              … method protocols that can be extended to other academic research centers. Final Outcome Proteins PINK1 and parkin …

… glucosylceramide and glucosylsphingosine. Our research interest is understanding the mechanistic and … glycosphingolipids in specific brain regions contributes to early Parkinson’s onset and accelerated progression rates.  … lipid imbalances for Parkinson’s therapies.   Impact on Diagnosis/Treatment of Parkinson’s Disease: Results in …