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.

Related Keywords

Stanford University ,California ,United States ,Italy ,Arizona ,France ,San Francisco ,French ,Italian ,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 ,