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 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…
Translational Candidate A library of induced pluripotent stem cell-derived cardiomyocytes from healthy subjects as well as patients with common hereditary cardiac disorders Area of Impact Preclinical toxicity screening and drug…
Translational Candidate The digital patient organoid (DPO) assay is a microfluidic miniature organoid technology that allows high-throughput drug testing. Area of Impact Our functional DPO assay will guide personalized therapy…
Induced pluripotent stem (iPS) cells are cells derived from skin that closely resemble embryonic stem (ES) cells and can be coaxed into many different types of cells such as nerve…
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