A hNSC Development Candidate for Huntington’s Disease
Huntington’s disease (HD) is a devastating degenerative brain disease with at least a 1 in 10,000 prevalence that inevitably leads to death. These numbers do not fully reflect the large…
Pre-clinical development of gene correction therapy of hematopoietic stem cells for SCID-X1
Severe combined immunodeficiency caused by mutations in the IL2RG gene on the x-chromosome (SCID-X1 or "bubble boy disease") is a devastating genetic disease that results in boys not being able…
Placental Stem Cells for the In Utero Treatment of Spina Bifida
Myelomeningocele – also known as spina bifida – is a devastating and costly defect that causes lifelong paralysis as well as bowel and bladder incontinence in newborns. It is one…
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…
CIRM Planning Grant in Leukemia
Leukemias are cancers of the blood forming cells that afflict both children and adults. Many drugs have been developed to treat leukemias and related diseases, but in many cases of…
{REDACTED} Cardiovascular Regenerative Team
Heart failure affects 5 million patients in the U.S., representing the most common cause of hospital admission and resulting in 300,000 deaths annually. Despite aggressive treatment with advanced pharmacotherapies and…
“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…
Genetically-modified neural stem cells for treatment of high-grade glioma
Our proposal details assembly of a multidisciplinary Disease Team whose goal will be to take into the clinic a novel and promising stem cell-based strategy for selectively targeting invasive tumor…
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,…
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