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Gene therapy may help children born with 'bubble boy' disease

Ten children with an especially rare and hard-to-treat form of "bubble boy" disease are living normal lives after receiving a new gene therapy approach, researchers say. ....

United States , Laverna Shorty , Morton Cowan , Stephen Gottschalk , David Vetter , Yun Pai , University Of California , Us National Cancer Institute , Jude Children Research Hospital In Memphis , Deficiency Foundation , San Francisco Benioff Children , New England Journal , Sung Yun Pai , Cancer Institute , Jude Children , Research Hospital , Immune Deficiency Foundation ,

Gene therapy promising for children with "bubble boy" syndrome

As gene therapies for rare diseases find success, more cracks are appearing in the current regulatory and commercialization models. ....

Stanford University , United States , San Francisco , Anne Galy , Yun Pai , Donald Kohn , Los Angeles , Morton Cowan , John Travolta , David Vetter , Maria Grazia Roncarolo , University Of California , National Cancer Institute , Institute For Stem Cell Biology , Regenerative Medicine At Stanford University , Autologous Gene Therapy , New England Journal , Jennifer Puck , Sung Yun Pai , National Cancer , Technological Research , Genomic Therapy , Regenerative Medicine Advanced Therapy , Stem Cell Biology , Regenerative Medicine , Orchard Therapeutics ,