Program Type: Translation
STEM CELL GENE THERAPY FOR SICKLE CELL DISEASE
Sickle cell disease (SCD), which results from an inherited mutation in the hemoglobin gene that causes red blood cells to “sickle” under conditions of low oxygen, occurs with a frequency of 1/500 African-Americans, and is also common in Hispanic-Americans, who comprise up to 5% of SCD patients in California. The median survival based on 1991 […]
“Stem Cell Therapies for Heart Failure”
Our multi-institutional program is dedicated to the treatment of heart failure, which has become a leading cause of death in California and the U. S. The high death toll continues to climb despite many significant recent advances in the medical treatment of heart failure. The disease is due to a debilitating loss and/or dysfunction of […]
Huntington’s Disease Team
Huntington’s disease (HD) is a devastating degenerative brain disease with a 1 in 10,000 risk of having a mutation that inevitably leads to death. These numbers do not fully reflect the large societal and familial cost of HD, which requires extensive caregiving. HD has no effective treatment or cure and symptoms progress without stopping for […]
{REDACTED} Heart Disease Regenerative Medicine Team Planning Award
The present planning grant application lays the groundwork for a collaborative heart disease regenerative medicine team. We plan to develop sequential preclinical and clinical investigations directed at regenerative stem cell-based approaches to treating the following major cardiovascular diseases: heart attack and its sequelae; congestive heart failure; and heart block requiring a pacemaker. The basic foundation […]
CIRM Disease Team Planning Award
Children born with sickle cell anemia (SCA), caused by a genetic defect in hemoglobin, have severe anemia and damage to virtually all the body organs: the damage begins in infancy, and is frequently fatal by early adulthood. This is one of the most common inherited diseases in the world: because of California’s ethnic diversity it […]
Stem cells for neuroprotection of photoreceptors in retinitis pigmentosa
The targeted disease is retinitis pigmentosa (RP), a severe form of blindness that runs in families. This disease is not common, yet represents an attractive near term target for stem cell therapy for a number of reasons: 1) RP destroys the light detecting cells of the retina but generally leaves the rest of the visual […]
Stem Cell-Mediated Oncocidal Therapy of Primary & Metastatic Brain Tumors
Brain tumors (BTs) are incurable, whether they start in the brain or spread there from other sites (e.g., lung, colon, breast, skin). Indeed, the latter situation is even more common & often more frustrating – although we’ve made inroads in treating such non-neural cancers, once brain metastases are discovered, hope is largely abandoned. Current therapies […]
Develop a cell replacement therapy for Parkinson’s disease using human embryonic stem cells
Parkinson’s disease (PD) is a devastating movement disorder caused by the death of dopaminergic neurons (a type of neurons in the central nervous system) present in the midbrain. These neurons secrete dopamine (a signaling molecule) and are a critical component of the motor circuit that ensures movements are smooth and coordinated. All current treatments attempt […]
Stem Cell Therapy for AIDS- Disease Team Planning
RNA interference is a naturally occurring means to block the function of genes in our body. We propose that RNA interference can be used to block HIV-1 infection and its reproduction within the body. When RNA interference is introduced into a stem cell, its blocking activity will be present throughout the lifetime of the stem […]
Human Stem Cell Approaches to Alzheimers Disease Therapies
Alzheimer’s Disease (AD) is a major health problem in the U.S. with 10% of people over the age of 65 and 50% of people over the age of 85 afflicted with disease. In spite of the magnitude of the problem, however, we lack a compelling mechanistic understanding of AD. As a result, we have no […]