Media Coverage
She Was Born Without an Immune System. Gene Therapy Saved Her Life.
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The New York Times
Gene therapy helps Virginia 12-year-old battle rare ‘bubble boy' disease
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NBC4 Washington
Exposure to any germs could've killed her. 11 years later, she's living a normal life thanks to a gene therapy treatment.
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NBC News
These Patients Got the Cure. Then It Went Away.
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The New York Times
They didn't understand why their kids kept getting sick. After a rare diagnosis, a new gene therapy offered hope.
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CBS News
Gene therapy trial helps children with LAD-I
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The Medicine Maker
Inhalable gene therapies? Here’s how they could work
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The Medicine Maker
Good news: genical therapy has canceled the symptoms of a rare genetic disease in 9 children
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Evidence Network
UCLA team developing one-time, inhalable CF gene-editing treatment
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Cystic Fibrosis News Today