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Gene Therapy's Year

Gene therapy has burst back onto the scene this year to show that it could be used to treat disease, the Associated Press reports. The idea had first been pursued in the late 1990s, but patient's death put a damper on the field that now seems to be lifted, it adds.

Just this year, three gene therapies have been approved in the US. In August, the Food and Drug Administration approved Novartis' Kymriah for acute lymphoblastic leukemia and Kite Pharmaceutical's Yescarta was approved in October to treat large B-cell lymphoma, both CAR T-cell therapies. Earlier this month, Spark Therapeutics' Luxturna was approved to treat retinal dystrophy. In addition, there are other efforts underway to harness gene editing such as CRIPSR approaches as therapies.

"I am totally willing to use the 'C' word," the National Institutes of Health Director Francis Collins tells the AP about the potential of gene therapy.

The AP notes that the treatments so far are expensive and don't work for everyone. Still, it says Collins is hopeful that diseases like muscular dystrophy and sickle cell will soon be tackled in this way.

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