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…
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,…
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…
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…
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…
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…
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…
Stem Cell Therapy for Muscular Dystrophy
The goal of our team is to capitalize on the remarkable advances in stem cell biology to apply those advances to the treatment of human diseases, disorders, and injuries. We…
Development of a Cell Replacement Therapy Product for Insulin Dependent Diabetes
With a current prevalence of greater than 170 million individuals world-wide, diabetes has attained epidemic proportions. The widespread secondary complications this disease extract a relentless and costly toll on the…
Pluripotent Stem Cell–Based Therapy for Heart Disease
Five million people in the U.S. suffer with heart failure, resulting in ~60,000 deaths/year at a cost of $30 billion/year. Heart failure occurs when the heart is damaged and becomes…
