Disease Focus: Alzheimer's Disease
Human Stem-Cell Based Development of a Potent Alzheimer’s Drug Candidate
Over 6 million people in the US suffer from Alzheimer’s disease (AD). There are no drugs that prevent the death of nerve cells in AD, nor has any drug been identified that can stimulate nerve cell replacement in aged human brain. Importantly, even if nerve cells could be replaced, the toxic environment of the AD […]
Optimizing the differentiation and expansion of microglial progenitors from human pluripotent stem cells for the study and treatment of neurological disease.
Microglia are a type of immune cell within the brain that profoundly influence the development and progression of many neurological disorders. Microglia also inherently migrate toward areas of brain injury, making them excellent candidates for use in cell transplantation therapies. Despite the widely accepted importance of microglia in neurological disease, methods to produce microglia from […]
Elucidating pathways from hereditary Alzheimer mutations to pathological tau phenotypes
We propose to elucidate pathways of genes that lead from early causes to later defects in Alzheimer’s Disease (AD), which is common, fatal, and for which no effective disease-modifying drugs are available. Because no effective AD treatment is available or imminent, we propose to discover novel genetic pathways by screening purified human brain cells made […]
Collection of skin biopsies to prepare fibroblasts from patients with Alzheimer’s disease and cognitively healthy elderly controls
Alzheimer’s Disease (AD), the most common form of dementia in the elderly, affects over 5 million Americans. There are no treatments to slow progression or prevent AD. This reflects limitations in knowledge of mechanisms underlying AD, and in tools and models for early development and testing of treatment. Genetic breakthroughs related to early onset AD […]
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 there are over 5 million individuals suffering from AD, including approximately 600,000 Californians. Current treatments do not alter the disease course. The absence of effective […]
Stem cell based small molecule therapy for Alzheimer’s disease
Over 6 million people in the US suffer from AD. There are no drugs that prevent the death of nerve cells in AD, nor has any drug been identified that can stimulate their replacement. Even if nerve cells could be replaced, the toxic environment of the brain will kill them unless they are protected by […]
Identifying Drugs for Alzheimer’s Disease with Human Neurons Made From Human IPS cells
We propose to discover new drug candidates for Alzheimer’s Disease (AD), which is common, fatal, and for which no effective disease-modifying drugs are available. Because no effective AD treatment is available or imminent, we propose to discover novel candidates by screening purified human brain cells made from human reprogrammed stem cells (human IPS cells or […]
Neuroprotection to treat Alzheimer’s: a new paradigm using human central nervous system cells
Alzheimer’s disease (AD) is an incurable disorder that affects memory, social interaction and the ability to perform everyday activities. In the USA alone, the number of AD patients aged 65 and older has surpassed 5 million and that number may triple by 2050. Annual health care costs have been estimated to exceed 172 billion dollars, […]
A CIRM Disease Team to Develop Allopregnanolone for Prevention and Treatment of Alzheimer’s Disease
Alzheimer’s disease (AD) is now a nation-wide epidemic and California is at the epicenter of the epidemic. One-tenth of all people in the United States diagnosed with AD live in California. In the US, 5.4 million people have AD and another American develops AD every 69 seconds. No therapeutic strategies exist to prevent or treat […]
Developing a method for rapid identification of high-quality disease specific hIPSC lines
Elucidating how genetic variation contributes to disease susceptibility and drug response requires human Induced Pluripotent Stem Cell (hIPSC) lines from many human patients. Yet, current methods of hIPSC generation are labor-intensive and expensive. Thus, a cost-effective, non-labor intensive set of methods for hIPSC generation and characterization is essential to bring the translational potential of hIPSC […]