Stem Cell Gene Therapy Restores Immune System in Kids with SCID

Researchers at UCLA have developed a stem cell gene therapy treatment for children born with the rare, life-threatening condition ADA-deficient SCID, often referred to as Bubble Baby disease. Children born with this condition are without a functioning immune system and are kept in controlled and isolated environments because exposure to common illnesses or infections can be lethal. We spoke to the treatment’s developer Donald Kohn of the UCLA Eli and Edythe Broad Center of Regenerative Medicine about the stem cell gene therapy, the promising results he’s had restoring normal immune function in clinical trial patients, and why this approach holds promise in other rare conditions.