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19 Feb 2015
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Gene Therapy Using Stem Cells for Efficient SCID Treatment

Researchers from UCLA team (University of California, Los Angeles) have introduced a successful gene therapy for SCID or bubble baby disease using bone marrow stem cells. Severe Combined Immunodeficiency (SCID) is a genetic disorder that affects the white blood cells responsible for the immune system in the body. Babies born with SCID are prone to all kinds of infection; even a common cold can be fatal. The children affected by SCID have no immune response and usually die within one year, if not treated.

The usual treatment method involves bone marrow transplant from matching donors. The patients can undergo a safe bone marrow transplant only if they have a sibling with matched HLA typing but most babies don’t. Other transplants include bone marrow from parents, unrelated donors and cord blood but these options involve risks. To overcome the risk, Dr. Donald Kohn and team at UCLA’s Human Gene Medicine Program developed a treatment strategy using the patient’s own bone marrow stem cells.

In this strategy, the bone marrow stem cells are isolated from the baby and the missing gene, ADA is inserted into the stem cells. The cells are injected back into the body which will restore the ability of cells to build a healthy immune system, thereby protecting the baby from germs and infection. There is no risk of rejection as the stem cells are retrieved from the patient’s own bone marrow. Dr. Kohn mentioned that it took over two decades of clinical trials to check the limitations and arrive at an improved strategy for successful treatment.

As the clinical trial is a success with all its participants being cured, the team is planning to get FDA approval; this way, all SCID infants can avail this treatment from hospitals. Recently, California Institute for Regenerative Medicine awarded Dr. Kohn and his team at UCLA with $13 million to support the clinical trial that involves the application of this strategy in the treatment of sickle cell disease.

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