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Bubble Boy Gene Therapy Utilizes Stem Cells

Posted by on Jan 8, 2016 1:31:00 PM

Scientists from the National Institute of Allergy and Infectious Diseases have developed a gene therapy utilizing stem cells, which restores immunity to patients with SCID-X1, also referred to as “Bubble Boy” disease. This genetic disorder prevents immune cells from developing properly, leaving patients prone to potentially fatal infections.

Transplantation of hematopoietic (blood-forming) stem cells, ideally from a genetically matched sibling donor, is the gold standard, lifesaving treatment for infants with SCID-X1. Children without a matched sibling often receive stem cells from a parent, which only partially restores immunity.

The future of treating immune-system-related genetic disorders will rely on gene therapies, which use bioengineered stem cells like those used in the study, rather than relying on donor cells. The success of these stem cell treatments is directly related to the use of autologous cells (the patient’s own), because the chance of rejection – a potentially life-threatening complication of treatment—is greatly limited.

The success of stem cell therapies for autoimmune disorders is an important area of research, as evidenced by the increasing practice autologous stem cell banking, which provides donor-based material for future therapies. As personalized regenerative medicine advances, autologous stem cells will become an invaluable resource to families and individuals who seek to ensure that they will have access to the best in medical care should the need for it arise. One of the most convenient and affordable sources for recovering and banking your own stem cells is dental stem cells. To learn more about how to bank dental stem cells for use in future therapies and treatments, visit StemSave or call 877-783-6728 (877-StemSave) today.



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Topics: stemsaveblog, genetic disease, gene therapy, hematopoietic stem cells, autoimmune disease, transplant, regenerative medicine


AAOMS - American Association of Oral and Maxillofacial Surgeons


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