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This boy was born without an immune system. Gene therapy rebuilt it.

TUBA CITY, Ariz. - Hataałii Tiisyatonii "HT" Begay, a Native American boy who is Diné, or Navajo, rolls toy trucks around in the dirt. He chases chickens into their coop. He pauses to watch his dad deftly gut a sheep for a ceremonial feast. Then he climbs onto a picnic table with his brother and cousins, chanting "Lava!" while pretending the ground has turned into molten rock. To his family, HT is a typical 5-year-old - a smiling, curious, busy blur who roams across his family's ranch, where the ....

San Francisco , United States , Tuba City , Grand Canyon , Morton Cowan , Brandon Jay Nez , Dana Hedgpeth , Courtney Nez , Darren Shorty , Laverna Shorty , Karen Yazzie , Brittney Nez , Donald Kohn , Peter Marks , University Of Colorado , University Of California At San Francisco , Translational Sciences , University Of Colorado Anschutz Medical Campus , National Center , Drug Administration , Division Of Rare Diseases Research Innovation , University Of California At Los Angeles , Native American , Navajo Nation , Phoenix Children , San Francisco Benioff Children ,

SCID: Gene therapy restored immune system in children with rare disorder

Ten children with the rare condition Artemis-deficient severe combined immunodeficiency had their immune systems either partially or fully restored with gene-replacement therapy ....

United States , New York , San Francisco , Morton Cowan , Vincent Bonagura , University Of California , Feinstein Institutes For Medical Research , Without Artemis , Jennifer Puck , Feinstein Institutes , Medical Research , New England Journal , Gene Therapy ,