Triplet Repeat Instability in Human iPSCs
Over twenty human genetic diseases are caused by expansion of simple DNA sequences composed of repeats of three nucleotides (such as CAG, CTG, CGG and GAA) within essential genes. These…
Neural and general splicing factors control self-renewal, neural survival and differentiation
Human embryonic and patient-specific induced pluripotent stem cells have the remarkable capacity to differentiate into many cell-types, including neurons, thus enabling the modeling of human neurological diseases in vitro, and…
Generation and characterization of corticospinal neurons from human embryonic stem cells
A major goal of stem cell research is to generate various functional human cell types that can be used to better understand how these cells work and to use them…
Understanding the role of LRRK2 in iPSC cell models of Parkinson’s Disease
The goal of this research is to utilize novel research tools to investigate the molecular mechanisms that cause Parkinson’s disease (PD). The proposed work builds on previous funding from CIRM…
Induced Pluripotent Stem Cells for Tissue Regeneration
Induced pluripotent stem cells (iPSCs) have tremendous potential for patient-specific cell therapies, which bypasses immune rejection issues and ethical concerns for embryonic stem cells (ESCs). However, to fully harness the…
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…
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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