Gene therapy trial shows promise for treating ‘bubble boy’ syndrome. Now comes the hard part
H.T. Begay is a happy kid. He’s smiley, silly, and definitely trying to make you laugh. The four-year-old’s two neat braids of dark hair wave behind him as he runs amongst the dust, dogs, and sheep near his family’s ranch on their Navajo reservation in Arizona.
On a table inside the family’s sweat lodge is a little altar. Next to a pair of tiny baby booties is a certificate that reads, “The first patient in the world to receive Autologous Gene Therapy for Artemis-deficient SCID 06/23/2018.”
