CRISPR/dCas9 mutant targeting SNCA promoter for downregulation of alpha-synuclein expression as a novel therapeutic approach for Parkinson’s disease

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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…

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Autologous cell therapy for Parkinson’s disease using iPSC-derived DA neurons

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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 linesCharacterize functionality of patient…

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A new phenotypic screening platform that identifies biologically-relevant targets and lead compounds for the treatment of Parkinson’s disease

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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…

Continue ReadingA new phenotypic screening platform that identifies biologically-relevant targets and lead compounds for the treatment of Parkinson’s disease

Reverse transcriptase inhibitors as a novel therapeutic approach for neurological autoimmune disorders

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Research Objective We found that approved anti-retroviral drugs could stop inflammation and block neurodegeneration. We propose to validate the re-purpose efficacy of these clinically-approved retroviral drugs. Impact We have identified…

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