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…
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…
The intestine performs the essential function of absorbing food and water into the body. Without a functional intestine, children and adults cannot eat normal meals, and these patients depend on…
The intestine performs the essential function of absorbing food and water into the body. Without a functional intestine, children and adults cannot eat normal meals, and these patients depend on…
Sickle cell disease (SCD)results from an inherited mutation in the hemoglobin gene that causes red blood cells to "sickle" under conditions of low oxygen. It occurs with a frequency of…
β-thalassemia is a genetic disease caused by diverse mutations of the β-globin gene that lead to profoundly reduced red blood cell (RBC) development. The unmet medical need in transfusion-dependent β-thalassemia…
Duchenne muscular dystrophy (DMD) affects 1 in every 3,500 boys worldwide. DMD is caused by mutations in the gene encoding dystrophin, a protein key to muscle health. DMD patients are…
Liver transplantation (LT) has been used to treat a variety of liver diseases. Within hours after birth, neonates can present with disorders of the urea cycle (UCDs), the critical metabolic…
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