This study will use Ataxia-Telangiectasia (A-T), an early-onset inherited neurodegenerative disease of children, as a model to study the mechanisms leading to cerebellar neurodegeneration and to develop a drug that…
Parkinson’s Disease (PD) is the most common neurodegenerative movement disorder. It is characterized by motor impairment such as slowness of movements, shaking and gait disturbances. Age is the most consistent…
The ability to convert human skin cells to induced pluripotent stem cells (IPSCs) represents a seminal break-through in stem cell biology. This advance effectively circumvents the problem of immune rejection…
The clinical application of cell replacement therapy in the US is dependent on the FDA's approval, and the primary objective of the FDA is to protect patients from unsafe drugs…
Human embryonic stem cells (hESC), and other related pluripotent stem cells, have great potential as starting material for the manufacture of curative cell therapies. This is primarily for two reasons.…
Research Objective We focus on hiPSCs pluripotency and neurodifferentiation to develop a novel framework to allow simultaneous identification of multiple interactions between proteins and between proteins and the genome Impact…
Research Objective Validating the cardiac MPS as tool to predict cardiac toxicity, including modeling drug-drug interaction of polytherapy for the treatment of SARS-CoV-2. Impact Our cardiac MPS will identify polytherapy…
Research Objective This study will use high throughput single-cell capture and next-generation sequencing technologies to resolve the composition of an emerging neuronal cell therapy product. Impact If successful, this study…
Research Objective We are developing a durable patient-specific dopamine neuron replacement therapy for Parkinson's disease Impact Parkinson's disease is diagnosed when half of the dopamine (DA) neurons are lost from…
Research Objective Development of a screen using inner ear sensory hair cell-like cells made by direct lineage reprogramming, for discovering drugs to ameliorate hearing loss during cancer chemotherapy. Impact Hearing…
