Disease Focus: Neurological Disorders
Immunization strategies to prevent Zika viral congenital eye and brain disease
Research Objective Our objective is to utilize human iPSC-derived neural and ocular cells to identify growth attenuated and non-pathogenic Zika virus vaccine candidates that can prevent congenital ZIKV disease. Impact Currently, there are no therapies or vaccines available against ZIKV for human use. The human iPSC technology provides a unique opportunity to test the growth […]
Discovery of therapeutics for Huntington’s Disease
Research Objective The objective of the proposed research is to perform 3 independent hESC-based screens to identify drug candidates for Huntington’s Disease. Impact There are currently no effective treatments for HD. Combination of human isogenic HD-mutants, novel tools and technology will provide therapeutic solutions for this neurodegenerative orphan disease. Major Proposed Activities Screening of 2,000 […]
A tool for rapid development of clinical-grade protocols for dopaminergic neuronal differentiation of Parkinson’s Disease patient-derived iPSCs
Research Objective Develop a tool that facilitates rapid, cost effective development of optimized GMP-grade hPSC differentiation into functional DA neurons and apply this device to a cohort of PD patient-derived iPSCs. Impact Creating GMP-grade, functionally consistent phenotypes for DA neurons from each patient will significantly increase the likelihood of stem cell-derived DA neuron-based therapy for […]
A treatment for Zika virus infection and neuroprotection efficacy
Research Objective We propose to determine the impact of the Zika virus during human neurodevelopment and to test a FDA-approved therapeutic candidate to treat Zika infection. Impact A drug to treat/cure Zika infection and for neuroprotection. Major Proposed Activities To determine the molecular and cellular alterations caused by the Zika virus in the human developing […]
CRISPR/dCas9 mutant targeting SNCA promoter for downregulation of alpha-synuclein expression as a novel therapeutic approach for Parkinson’s disease
Research Objective Discovery of a novel therapeutic candidate for Parkinson’s disease which modifies gene expression using human stem cell-derived neurons to halt the neurodegenerative disease process. Impact Stopping the neurodegenerative process of Parkinson’s disease is a critical unmet medical need. Our approach is based on novel gene engineering technology that modifies expression of key target […]
hNSC-mediated delivery of ApiCCT1 as a candidate therapeutic for Huntington’s disease
Research Objective The therapeutic candidate is a human Neural Stem Cell that secretes a protein, ApiCCT1, that aids in the prevention of disease phenotypes, for application in treatment of Huntington's disease (HD). Impact No treatment currently exists that can slow or prevent the unrelenting progression of Huntington’s disease, a devastating brain disease, therefore a completely […]
Autologous cell therapy for Parkinson’s disease using iPSC-derived DA neurons
Research Objective Autologous human dopaminergic neurons derived from patient-specific induced pluripotent stem cells Impact Parkinson's disease Major Proposed Activities Characterize differentiation from all 10 patient cell lines Characterize functionality of patient neurons matured in vitro Immunogenicity assessment Cryopreservation feasibility testing Investigate dose response in vivo Detect dopamine release in vivo Thousands of Californians suffer from […]
MSC delivery of an artificial transcription factor to the brain as a treatment for Angelman Syndrome
Research Objective Mesenchymal stem cells will be used to deliver an artificial transcription factor to neurons in the brain to treat a genetic disease. Impact It could lead directly to a treatment for Angelman Syndrome, but the approach could be used to alter gene expression in almost any brain disorder. It could overcome the brain […]
Scalable, Defined Production of Oligodendrocyte Precursor Cells to Treat Neural Disease and Injury
Research Objective The goal of this proposal is to develop an optimized, scalable process to manufacture high quality oligodendrocyte precursor cells (OPCs) from human pluripotent stem cells for treating human disease. Impact OPCs have therapeutic potential for spinal cord injury, restoration of cognitive function after cancer radiation therapy, inherited demyelinating disease, and potentially multiple sclerosis. […]
A new phenotypic screening platform that identifies biologically-relevant targets and lead compounds for the treatment of Parkinson’s disease
Research Objective Demonstrate that our HitFinder™ library can be screened for phenotypic changes in A53T-IPSC-derived dopaminergic neurons and use a secondary handle to identify the targets responsible. Impact This technology combines phenotypic screening and target-ID eliminating the need to bias assays and/or screening libraries permitting application directly in iPSC-derived cells. Major Proposed Activities Prepare screening […]