UCLA researcher pioneers gene therapy cure for ‘Bubble Baby’ disease

UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with a life-threatening condition called adenosine deaminase–deficient severe combined immunodeficiency, or ADA-deficient SCID. Often called Bubble Baby disease, the condition can be fatal within the first year of life if left untreated. The groundbreaking treatment was developed by Dr. Donald Kohn, a renowned stem cell researcher and member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research . Kohn’s breakthrough was developed over three decades of research aimed at creating a gene therapy that safely restores the immune systems of children with ADA-deficient SCID using the patients’ own cells with no side effects. To date, 18 children with SCID have been cured of the disease after receiving the therapy in clinical trials at UCLA and the National Institutes of Health. “All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy,” said Kohn, a professor of pediatrics and of microbiology, immunology and molecular genetics in the UCLA College. “Instead they are all thriving with fully functioning immune systems.” Children born with SCID are kept in controlled, isolated environments because without an immune system, ordinary illness and infection could be lethal.   “Other current options for treating ADA-deficient SCID ...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news

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