Therapeutic/Technology: Therapeutic Approach
iPS Cell-Based Treatment of Dominant Dystrophic Epidermolysis Bullosa
Genetic skin diseases constitute a diverse group of several hundred diseases that affect up to 2% of the population and include common disease such as psoriasis, atopic dermatitis, and wound healing. Patients with one genetic disease, dystrophic Epidermolysis bullosa (EB), lack a normal collagen VII (COL7A1) gene and suffer from debilitating blistering and scarring that […]
Development of Therapeutic Antibodies Targeting Human Acute Myeloid Leukemia Stem Cells
Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow that is rapidly fatal within months if untreated. Even with aggressive treatment, including chemotherapy and bone marrow transplantation, five-year overall survival rates range between 30-40%. Evidence indicates that not all cells in this cancer are the same, and that there is a […]
Therapeutic Opportunities To Target Tumor Initiating Cells in Solid Tumors
Cancer is a major cause of human death worldwide. The vast majority of cancer patients suffer from solid tumors whose growth destroys vital organs. We propose to develop novel therapeutic drugs that target solid tumors affecting the brain, colon and ovaries. These cancers account for a significant proportion of currently intractable solid malignancies. Scientists have […]
ZINC FINGER NUCLEASE-BASED STEM CELL THERAPY FOR AIDS
Some years ago it was discovered that patients homozygous for a natural mutation (the Δ32 mutation) in the CCR5 gene are generally resistant to HIV infection by blocking virus entry to a cell. Building on this observation, a study published in 2009 reported a potential “cure” in an AIDS patient with leukemia after receiving a […]
Stem cell based treatment strategy for Age-related Macular Degeneration (AMD)
Retinal degeneration represents a group of blinding diseases that are increasingly impacting the health and well being of Californians. It is estimated that by 2020, over 450,000 Californians will suffer from vision loss or blindness due to the age-related macular degeneration (AMD), the most common cause of retinal degeneration diseases in the elderly. AMD is […]
Development of Highly Active Anti-Leukemia Stem Cell Therapy (HALT)
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. These drugs are often effective when first given, but in many cases of adult leukemia, the disease returns in a form that is not curable, causing disability and eventual death. […]
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 […]