UCLA Researchers Announce Gene Therapy Cure for 18 oeBubble Baby Patients

A team led by Dr. Donald Kohn, a stem cell researcher at the university's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research in Los Angeles, developed the breakthrough that cured 18 children who had adenosine deaminase -deficient severe combined immunodeficiency . "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, instead they are all thriving with fully functioning immune systems," Kohn, a professor of pediatrics and of microbiology, immunology and molecular genetics in the life sciences at UCLA, said in a statement.

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