Disease Focus: Parkinson's Disease

Directed Evolution of Novel AAV Variants for Enhanced Gene Targeting in Pluripotent Human Stem Cells and Investigation of Dopaminergic Neuron Differentiation

Human embryonic stem cells (hESCs) and induced pluripotent stem (iPS) cells have considerable potential as sources of differentiated cells for numerous biomedical applications. The ability to introduce targeted changes into the DNA of these cells – a process known as gene targeting – would have very broad implications. For example, mutations could readily be introduced […]

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 […]

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 […]

Molecular and Cellular Transitions from ES Cells to Mature Functioning Human Neurons

Human embryonic stem cells (hESCs) are pluripotent entities, capable of generating a whole-body spectrum of distinct cell types. We have developmental procedures for inducing hESCs to develop into pure populations of human neural stem cells (hNS), a step required for generating authentic mature human neurons. Several protocols have currently been developed to differentiate hESCs to […]

Modeling Parkinson’s Disease Using Human Embryonic Stem Cells

Parkinson’s disease (PD) is the most frequent neurodegenerative movement disorder caused by damage of dopamine-producing nerve cells (DA neuron) in patient brain. The main symptoms of PD are age-dependent tremors (shakiness). There is no cure for PD despite administration of levodopa can help to control symptoms. Most of PD cases are sporadic in the general […]

Optimization of guidance response in human embryonic stem cell derived midbrain dopaminergic neurons in development and disease

A promising approach to alleviating the symptoms of Parkinson’s disease is to transplant healthy dopaminergic neurons into the brains of these patients. Due to the large number of transplant neurons required for each patient and the difficulty in obtaining these neurons from human tissue, the most viable transplantation strategy will utilize not fetal dopaminergic neurons […]

Identifying small molecules that stimulate the differentiation of hESCs into dopamine-producing neurons

In this application, we propose to identify small molecule compounds that can stimulate human embryonic stem cells to become dopamine-producing neurons. These neurons degenerate in Parkinson’s disease, and currently have very limited availability, thus hindering the cell replacement therapy for treating Parkinson’s disease. Our proposed research, if successful, will lead to the identification of small […]

Identification and characterization of human ES-derived DA neuronal subtypes

Parkinson’s disease (PD) is a neurodegenerative movement disorder that affects 1 in 100 people over the age of 60, one million people in the US and six million worldwide. Patients show a resting tremor, slowness of movement (bradykinesia), postural instability and rigidity. Parkinson’s disease results primarily from the loss of neurons deep in the middle […]

Lorenz Studer, Winner of the 2017 Ogawa-Yamanaka Stem Cell Prize

Don’t miss our video interview with Dr. Lorenz Studer, esteemed stem cell scientist at Sloan Kettering and winner of the 2017 Gladstone Institutes Ogawa-Yamanaka Stem Cell Prize. Read our blog for more details about the Dr. Studer’s research

Lorenz Studer, Winner of the 2017 Ogawa-Yamanaka Stem Cell Prize

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