In Groundbreaking First, Doctors Say They Have Cured Several Infants of ‘Bubble Boy’ Disease

For the first time ever, scientists have used gene therapy to cure several young patients of “bubble boy” disease, a rare genetic defect that leaves children with an ineffective or nonexistent immune system.

The “bubble boy” name is a reference to how the patients, who usually die early in life if left untreated, are often forced to live in sterile environments.

Now, however, gene therapy developed at St. Jude Children’s Research Hospital has cured 10 infants born with X-linked severe combined immunodeficiency (SCID-X1), the most common type of the rare, life-threatening genetic condition.

The children are currently producing functional immune cells, including T cells, B cells and natural killer (NK) cells, for the first time.


https://www.goodnewsnetwork.org/doctors-cure-infants-of-bubble-boy-disease/