-
Functional Analysis of LRRK2: Focus on Autophagy
… original grant was to utilize pre-clinical and human model systems to better understand LRRK biology. The original aims … LRRK2 KO model with respect to the dopaminergic system and 3) create human lines containing PD associated … the role of autophagy in basic LRRK2 biology, in the immune system, and in mitochondrial quality control. Importance of …
-
CRISPRi/dCas9 Downregulation of Alpha-synuclein as a Novel Gene Therapy
… to alter a particular location in the genome. The CRISPR system penetrates the cells through a safe delivery system and will only change the expression of the SNCA gene … study will optimize delivery and the design of the CRISPR system in pre-clinical models of tParkinson’s disease and …
-
Generation of Transplantable Dopamine Neurons from Human Emryonic Stem Cells
… all cell types in our body. We have established a culture system to induce human ES cells to give rise to precursors … of brain and spinal cord cells. We will create a culture system that allows the neural precursors to generate … dopamine neurons. This way, we will optimize the culture system to generate maximal number of transplantable dopamine …
-
Arizona Parkinson's Disease Consortium: Predictors of Parkinson’s Disease and Parkinson’s Disease-Dementia
… sites for diagnosing PD, test current pathological staging systems for PD and survey the spinal cord and peripheral nervous system for the distribution of Lewy body-related pathology. … in PD, publication of an improved pathological staging system for PD and the first detailed mapping of Lewy …
-
Vikram Khurana, MD, PhD
… to understanding alpha-synuclein biology using model systems ranging from yeast to human stem cells, advancing … disease (PD). His group developed experimental and systems biology approaches to stratify patients with …
-
Anne Weissbach, MD
… Anne Weissbach is a clinician-scientist at the Institute of Systems Motor Science and the Institute of Neurogenetics at … at the University of Toronto, Canada and the Institute of Systems Motor Science of Alexander Münchau at the UKSH …