Modeling disease in human embryonic stem cells using new genetic tools
The use of stem cells or stem cell-derived cells to treat disease is one important goal of stem cell research. A second, important use for stem cells is the creation…
Role of the NMD RNA Decay Pathway in Maintaining the Stem-Like State
A subset of intellectual disability cases in humans are caused by mutations in an X-linked gene essential for a quality control mechanism called nonsense-mediated RNA decay (NMD). Patients with mutations…
Modeling Alexander disease using patient-specific induced pluripotent stem cells
Alexander disease (AxD) is a devastating childhood disease that affects neural development and causes mental retardation, seizures and spasticity. The most common form of AxD occurs during the first two…
Common molecular mechanisms in neurodegenerative diseases using patient based iPSC neurons
A major medical problem in CA is the growing population of individuals with neurodegenerative diseases, including Parkinson’s (PD) and Huntington’s (HD) disease. These diseases affect millions of people, sometimes during…
Stem Cell Mechanisms Governing Discrete Waves of Gliogenesis in the Childhood Brain
White matter is the infrastructure of the brain, providing conduits for communication between neural regions. White matter continues to mature from birth until early adulthood, particularly in regions of brain…
Stem cell models to analyze the role of mutated C9ORF72 in neurodegeneration
Amyotrophic lateral sclerosis (ALS) is an idiopathic adult-onset degenerative disease characterized by progressive weakness from loss of upper and lower motor neurons. Onset is insidious, progression is essentially linear, and…
Progenitor Cells Secreting GDNF for the Treatment of ALS
This project aims to use a powerful combined neural progenitor cell and growth factor approach to treat patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s Disease). ALS is a…
Restoration of memory in Alzheimer’s disease: a new paradigm using neural stem cell therapy
Alzheimer’s disease (AD), the leading cause of dementia, results in profound loss of memory and cognitive function, and ultimately death. In the US, someone develops AD every 69 seconds and…
MSC engineered to produce BDNF for the treatment of Huntington’s disease
One in every ten thousand people in the USA has Huntington's disease, and it impacts many more. Multiple generations within a family can inherit the disease, resulting in escalating health…
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