
Barbara Riles for UCSF
History making gene therapy makes childhood possible
Within days of his birth, Hataalii Tiisyatonii (“HT”) was diagnosed with Artemis-SCID, a serious form of primary immunodeficiency, also known as “bubble baby” disease. Children with Artemis-SCID lack a functioning immune system, making them much more susceptible to infections. Many children with the disease don’t live past their first birthday. When a bone marrow transplant – the standard treatment for the disease – wasn’t viable for HT, University of California, San Francisco pediatricians thought their clinical trial might be the solution. HT became the first child in the world with Artemis-SCID to receive gene-correction therapy. The trial and procedure were successful and after a long recovery, HT is now back home in Arizona loving life on his family’s ranch.