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Gene therapy comes of age

12 Jan 2018

Abstract excerpt

After almost 30 years of promise tempered by setbacks, gene therapies are rapidly becoming a critical component of the therapeutic armamentarium for a variety of inherited and acquired human diseases. Gene therapies for inherited immune disorders, hemophilia, eye and neurodegenerative disorders, and lymphoid cancers recently progressed to approved drug status in the United States and Europe, or are anticipated to...

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PMID: 29326244
DOI: 10.1126/science.aan4672

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