Elucidating Molecular Basis of Hypertrophic Cardiomyopathy with Human Induced Pluripotent Stem Cells
Familial hypertrophic cardiomyopathy (HCM) is the leading cause of sudden cardiac death in young people, including trained athletes, and is the most common inherited heart defect. Until now, studies in…
Homologous recombination in human pluripotent stem cells using adeno-associated virus.
Since their discovery in 1998, human embryonic stem cells (hESCs) have been considered to hold great potential for the treatment of many currently incurable diseases. Possibly the most exciting application…
Cellular tools to study brain diseases affecting synaptic transmission
There is a group of brain diseases that are caused by functional abnormalities. The brains of patients afflicted with these diseases which include autism spectrum disorders, schizophrenia, depression, and mania…
Antibody tools to deplete or isolate teratogenic, cardiac, and blood stem cells from hESCs
Purity is as important for cell-based therapies as it is for treatments based on small-molecule drugs or biologics. Pluripotent stem cells possess two properties: they are capable of self regeneration…
Tri-resolution Visualization System for Stem Cells and Tissue Regeneration Monitoring
The 3D imaging techniques of CT and MRI have virtually eliminated the need for exploratory surgery – a procedure which was common in difficult cases just 20-30 years ago. Not…
Use of iPS cells (iPSCs) to develop novels tools for the treatment of spinal muscular atrophy.
Spinal Muscular Atrophy (SMA) is one of the most common lethal genetic diseases in children. One in thirty five people carry a mutation in a gene called survival of motor…
Engineering Defined and Scaleable Systems for Dopaminergic Neuron Differentiation of hPSCs
Human pluripotent stem cells (hPSC) have the capacity to differentiate into every cell in the adult body, and they are thus a highly promising source of differentiated cells for the…
Development of Single Cell MRI Technology using Genetically-Encoded Iron-Based Reporters
Clinical application of cell transplantation therapy requires a means of non-invasively monitoring these cells in the patient. Several imaging modalities, including MRI, bioluminescence imaging, and positron emission tomography have been…
Editing of Parkinson’s disease mutation in patient-derived iPSCs by zinc-finger nucleases
The goal of this proposal is to establish a novel research tool to explore the molecular basis of Parkinson’s disease (PD) - a critical step toward the development of new…
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