Program Type: Translation
Development of a Chondrogenic Drug Candidate Targeting Cartilage-residing Mesenchymal Stem Cells for the Treatment of Osteoarthritis
Osteoarthritis (OA) is the most prevalent musculoskeletal disease affecting nearly 27 million people in the United States, and is the leading cause of chronic disability in the United States. Current therapeutic options are limited to pain or symptom-modifying drugs and joint replacement surgery; no disease-modifying drugs are approved for clinical use. OA is characterized by […]
Embryonic Stem Cell-Derived Chondroprogenitor Cells to Repair Osteochondral Defects
Surgical approaches to the treatment of focal cartilage defects can be classified into repair, replacement, and regeneration therapies. Marrow stimulation procedures such as microfracture result in a repair tissue that is predominantly fibrocartilaginous in nature, which is mechanically less durable than articular cartilage and survives on average 7 years before requiring another procedure. Osteochondral grafting […]
Scaffold for dermal regeneration containing pre-conditioned mesenchymal stem cells to heal chronic diabetic wounds
The goal of our CIRM-funded Early Translational (ETA) grant was to engineer a product to improve healing in diabetic foot ulcers, a devastating consequence of diabetes that occurs in about 25% of all diabetic patients and is responsible for most leg or foot amputations. More than 6 million people in the US and up to […]
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 societal and familial cost of HD, which requires extensive care-giving. HD has no effective treatment or cure and symptoms unstoppably progress for 15-20 years, with […]
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 to form an immune system. If they are exposed to the environment for even a short period of time they can get infections that a […]
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 of the most common birth defects worldwide, with four children in the United States born with spina bifida every day. Spina bifida affects the physical, […]
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 identified that can stimulate nerve cell replacement in aged human brain. Importantly, even if nerve cells could be replaced, the toxic environment of the AD […]
{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 implantable devices, the 5-year survival is only 50%. Cardiac transplantation is limited to 2,000 patients per year due to the lack of suitable donors. Therefore, […]
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 adult leukemia, the diseases are not curable, and cause disability and eventual death. More than in other cancers, scientists understand the exact molecular changes in […]
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 cells in high-grade glioma. These tumor cells form small foci scattered throughout the brain that are resistant to standard treatments and are the tumor in […]