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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 statesLaverna shortyMorton cowanStephen gottschalkDavid vetterYun paiUniversity of californiaUs national cancer instituteJude children research hospital in memphisDeficiency foundationSan francisco benioff childrenNew england journalSung yun paiCancer instituteJude childrenResearch hospital

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 universityUnited statesSan franciscoAnne galyYun paiDonald kohnLos angelesMorton cowanJohn travoltaDavid vetterMaria grazia roncaroloUniversity of californiaNational cancer instituteInstitute for stem cell biologyRegenerative medicine at stanford universityAutologous gene therapy

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