UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with adenosine deaminase -deficient severe combined immunodeficiency , often called "Bubble Baby" disease, a life-threatening condition that if left untreated can be fatal within the first year of life. The groundbreaking treatment was developed by renowned stem cell researcher and UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member Dr. Donald Kohn, whose breakthrough was developed over three decades of research to create a gene therapy that safely restores immune systems in children with ADA-deficient SCID using the patient's own cells with no side effects.
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