Retinitis pigmentosa is a blinding eye disease that affects approximately 150,000 individuals in the US and 1.5 million people around the world. It is caused by the destruction of light-sensing…
Cystinosis is a rare disease that primarily affects children and young adults, and leads to premature death, usually in early adulthood. Patients inherit defective copies of a gene called CTNS,…
Transplantation of beta cells, contained in donor pancreatic islets, can reverse the symptoms of diabetes. However, due to a poor islet survival rate, transplants require islets from multiple donors. Since…
Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease caused by a mutation in a specific gene that affects the body's ability to combat infections. As a result, infants with…
Angiocrine Bioscience Inc. will use genetically engineered cells, derived from cord blood, to see if they can help alleviate or accelerate recovery from the toxic side effects of chemotherapy for…
Sangamo, Inc. is testing genetically engineered blood stem cells for the treatment of beta-thalassemia, a severe form of anemia caused by mutations in the hemoglobin gene. This genetic disorder requires…
UC San Francisco researchers aim to repair the damaged immune system of children born with severe combined immunodeficiency (SCID), a genetic blood disorder in which even a mild infection can…
Viral infection can lead to fatal complications in patients with weakened immune systems resulting from chemotherapy, bone marrow or cord blood transplant, and other forms of inherited or acquired disorders.…
Pulmonary arterial hypertension (PAH) is a progressive condition with no cure. Scientists at Cedars-Sinai Medical Center are using donor cells derived from the heart to reduce two hallmark symptoms of pulmonary…
Femoral head osteonecrosis (aka avascular necrosis) is a disease caused by loss of blood supply to the bone, leading to bone cell death, end stage hip arthritis and total hip…
