Stage of Program: Preclinical Research
Modeling Myocardial Therapy with Human Embryonic Stem Cells
Five million people in the U.S. suffer with heart failure, at a cost of $30 billion/year. Heart failure occurs when the heart is damaged and becomes unable to meet the demands placed on it. Unlike some tissues, heart muscle does not regenerate. Human embryonic stem cells grow and divide indefinitely while maintaining the potential to […]
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: […]
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 […]
Embryonic-Derived Neural Stem Cells for Treatment of Motor Sequelae following Sub-cortical Stroke
A stroke kills brain cells by interrupting blood flow. The most common “ischemic stroke” is due to blockage in blood flow from a clot or narrowing in an artery. Brain cells deprived of oxygen can die within minutes. The loss of physical and mental functions after stroke is often permanent and includes loss of movement, […]
Stem Cell-Derived Astrocyte Precursor Transplants in Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS), a lethal disease lacking effective treatments, is characterized by the loss of upper and lower motor neurons. 5-10% of ALS is familial, but the majority of ALS cases are sporadic with unknown causes. The lifetime risk is approximately 1 in 2000. This corresponds to ~30,000 affected individuals in the United States […]
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 […]
HPSC based therapy for HIV disease using RNAi to CCR5.
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 […]
Autologous cardiac-derived cells for advanced ischemic cardiomyopathy
The adult human heart contains small numbers of cardiac stem cells that are able to partially repair the heart following a heart attack or throughout the course of progressive heart failure. We have developed a method to isolate these cells and grow them to large numbers in the lab. Isolation begins with a minimally-invasive biopsy […]
Stem Cell-Mediated Oncocidal Gene Therapy of Glioblastoma (GBM)
Brain tumors (BTs) are incurable, whether they start in the brain or spread there from other sites. Despite advances in surgical, radiation, pharmacologic, and gene therapies, survival with a BT remains dismal. Current therapies are limited by their inability to reach widely disseminated tumor cells that become dispersed within normal brain structures. Interestingly, the therapeutic […]
Stem Cell-mediated Therapy for High-grade Glioma: Toward Phase I-II Clinical Trials
Despite aggressive multimodal therapy and advances in imaging, surgical and radiation techniques, malignant brain tumors (high-grade gliomas) remain incurable, with survival often measured in months. Treatment failure is largely attributable to the diffuse and invasive nature of these brain tumor cells, ineffective delivery of chemotherapeutic agents to tumor sites, and toxic side-effects to the body, […]