Disease Focus: Neurological Disorders
Derivation of Parkinson’s Disease Coded-Stem Cells (PD-SCs)
Parkinson’s disease (PD) is currently the most common neurodegenerative movement disorder, severely debilitating approximately 1-2% of the US population. The disease is caused by a selective loss of dopamine-producing neurons located in a specific region of the brain. This loss leads to significant motor function impairment and age-dependent tremors. Unfortunately there is currently no cure […]
Generation of clinical grade human iPS cells
The therapeutic use of stem cells depends on the availability of pluripotent cells that are not limited by technical, ethical or immunological considerations. The goal of this proposal is to develop and bank safe and well-characterized patient-specific pluripotent stem cell lines that can be used to study and potentially ameliorate human diseases. Several groups, including […]
Establishment of Frontotemporal Dementia Patient-Specific Induced Pluripotent Stem (iPS) Cell Lines with Defined Genetic Mutations
We propose to generate induced pluripotent stem (iPS) cells from skin cells derived from human subjects with frontotemporal dementia (FTD). FTD accounts for 15–20% of all dementia cases and, with newly identified genetic causes, is now recognized as the most common dementia in patients under 65 years of age. FTD patients suffer progressive neurodegeneration in […]
Development of Induced Pluripotent Stem Cells for Modeling Human Disease
Human embryonic stem cells (hESC) hold great promise in regenerative medicine and cell replacement therapies because of their unique ability to self-renew and their developmental potential to form all cell lineages in the body. Traditional techniques for generating hESC rely on surplus IVF embryos and are incompatible with the generation of genetically diverse, patient or […]
New Cell Lines for Huntington’s Disease
Huntington’s disease (HD) is a devastating neurodegenerative disease with a 1/10,000 disease risk that always leads to death. These numbers do not fully reflect the large societal and familial cost of HD, which requires extensive caregiving and has a 50% chance of passing the mutation to the next generation. Current treatments treat some symptoms but […]
hESC-Derived Motor Neurons For the Treatment of Cervical Spinal Cord Injury
Cervical spinal cord injuries result in a loss of upper limb function because the cells within the spinal cord that control upper limb muscles are destroyed. The goal of this research program is to create a renewable human source of these cells, to restore upper limb function in both acute and chronic spinal cord injuries. […]
MEF2C-Directed Neurogenesis From Human Embryonic Stem Cells
Understanding differentiation of human embryonic stem cells (hESCs) provides insight into early human development and will help directing hESC differentiation for future cell-based therapies of Parkinson’s disease, stroke and other neurodegenerative conditions. The PI’s laboratory was the first to clone and characterize the transcription factor MEF2C, a protein that can direct the orchestra of genes […]
Human stem cell derived oligodendrocytes for treatment of stroke and MS
Strokes that affect the nerves cells, i.e., “gray matter”, consistently receive the most attention. However, the kind of strokes that affecting the “wiring” of the brain, i.e., “white matter”, cause nearly as much disability. The most severe disability is caused when the stroke is in the wiring (axons) that connect the brain and spinal cord; […]
Epigenetic gene regulation during the differentiation of human embryonic stem cells: Impact on neural repair
Human embryonic stem cells (hESCs) have the potential to become all sorts of cells in human body including nerve cells. Moreover, hESCs can be expanded in culture plates into a large quantity, thus serving as an ideal source for cell transplantation in clinical use. However, the existing hESC lines are not fully characterized in terms […]
Spinal ischemic paraplegia: modulation by human embryonic stem cell implant.
schemia-induced paraplegia often combined with a qualitatively defined increase in muscle tone (i.e. spasticity and rigidity) is a serious complication associated with a temporary aortic cross-clamping ( a surgical procedure to repair an aortic aneurysm). In addition to spinal ischemic injury-induced spasticity and rigidity a significant population of patients with traumatic spinal injury develop a […]