Best of CASE District VII Award
 

Institution: University of California, San Francisco

Title of entry: How Gene Therapy Saved a Child from ‘Bubble Boy’ Disease

About this entry: “Bubble Boy Disease,” is a genetic disease that kills roughly half of those born with it early in their lives. Children with the disorder, also known as Artemis-SCID, are born without a functional immune system and are highly susceptible to infection. UCSF News tells the story of the first child in the world with Artemis-SCID to have his own stem cells harvested, gene-corrected, and re-implanted — rather than using the cells of a donor.

The treatment was pioneered by two UCSF researchers who’ve spent a lifetime working on SCID. The child, who goes by HT, is a Navajo descendant — a population at higher risk for the disease. HT’s journey, and that of his grandmother — who brought him to San Francisco so he could receive treatment for several months — is an inspiring tale of resilience, bravery, and trust.

To tell this story, we visited with HT, his grandmother, and UCSF physicians in San Francisco. And we traveled to Arizona to see HT readjusting to life on the reservation — playing in the dirt, caring for livestock, and enjoying his extended family. The article was the basis of our storytelling and a resource for news media.

Our article, published Dec. 21 to coincide with The New England Journal of Medicine’s publication of the research, showcases UCSF’s focus on innovative research leading to breakthroughs in health care. It also promotes UCSF's mission and reputation as an elite health sciences and research university that serves the most vulnerable populations.