Program Type: Discovery
The Dangers of Mitochondrial DNA Heteroplasmy in Stem Cells Created by Therapeutic Cloning
n therapeutic cloning, a patient’s cell is combined (fused) to an enucleated donated egg (oocyte) from an unrelated woman or from another animal. It is hoped that cellular factors in the egg cytoplasm will reprogram the patient’s cell nucleus making it capable of generating replacement cells for the patient’s body. Thus, if a patient is […]
Derivation of Inhibitory Nerve Cells from Human Embryonic Stem Cells
Parkinson’s disease (PD) is caused by degeneration of a specific population of dopamine-producing nerve cells in the brain and is chronic, progressive, and incurable. Loss of dopamine-containing cells results in profound physiological disturbances producing tremors, rigidity, and severe deterioration of gate and balance. In the United States, approximately 1.5 million people suffer with PD and […]
Engineering a Cardiovascular Tissue Graft from Human Embryonic Stem Cells
Cardiovascular disease (CVD) affects more than 71 million Americans and 1.7 million Californians. Recently, engineered cardiovascular tissue grafts, or “patches”, including one made from mouse embryonic stem cells (ESC), have shown promising results as a future therapy for CVD. Our overall goal is to extend these recent results to human ESC as follows. Aim 1: […]
Immunology of neural stem cell fate and function
One of the most difficult yet ultimately rewarding goals in stem cell research is to repair damaged neural systems with newly generated neurons. Our work examining neuronal integration and survival in the postnatal and adult brain shows that incoming neurons are uniquely and exquisitely sensitive to the immune response and inflammation that is always present […]
Chemical Genetic Approach to Production of hESC-derived Cardiomyocytes
Adult heart muscle cells retain negligible proliferative capacity and this underlies the inability of the heart to replace muscle cells that are lost to injury, such as infarct, and underlies progression to heart failure. To date, no stem cell therapiy has produced significant cardiomyocyte replacement. Instead, transplanted cells, if they persist at all, produce endothelial […]
Embryonic Stem Cell-Derived Therapies Targeting Cardiac Ischemic Disease
Cardiovascular disease (CVD) is the leading cause of death in the United States. Over one million Americans will suffer from a new or recurrent heart attacks this year and over 40 percent of those will die suddenly. In addition, about two-thirds of the patients develop congestive heart failure; and in people diagnosed with CHF, sudden […]
Using Human Embryonic Stem Cells to Understand and to Develop New Therapies for Alzheimer’s Disease
Alzheimer’s Disease (AD) is a progressive incurable disease that robs people of their memory and ability to think and reason. It is emotionally, and sometimes financially devastating to families that must cope when a parent or spouse develops AD. Unfortunately, however, we currently lack an understanding of Alzheimer’s Disease (AD) that is sufficient to drive […]
Functional Genomic Analysis of Chemically Defined Human Embryonic Stem Cells
Regenerative medicine holds the promise that tissues can be engineered in vitro and then transplanted into patients to treat debilitating diseases. Human Embryonic Stem Cells differentiate into a wide array of adult tissue types and are thought to be the best hope for future regenerative therapies. This grant has three main goals: 1. The creation […]
Derivation and characterization of human ES cells from FSHD embryos
Facioscapulohumeral muscular dystrophy (FSHD) is the third most common hereditary muscular dystrophy. It is autosomal dominant, meaning that if one of the parents has the disease, their children have a 50:50 chance of getting it, too. FSHD is characterized by progressive weakness and atrophy of facial, shoulder and upper arm musculature, which can spread to […]
Hair Cells and Spiral Ganglion Neuron Differentiation from Human Embryonic Stem Cells
Hair cells (HCs) convert sound and balance signals into electrical impulses in the inner ear, including the cochlea and the vestibular endorgans, with remarkable precision and sensitivity. Our long-term goal is to stimulate HC regeneration in human inner ears and to enable the functional innervations of HCs by neurons. Hair cells are terminally-differentiated cells. Once […]