The FDA is considering approving the first gene therapy for blindness. How does it work?

Friday, 13 October 2017, 05:33:31 PM. The one-time treatment could restore eyesight to thousands of people.
Share Tech & Science retinal dystrophy Blindness gene therapy FDA Updated | A Food and Drug Administration committee has voted that a gene therapy touted as a breakthrough for people with a particular genetic form of blindness is effective. A final FDA approval for the treatment, Luxturna, would be a landmark event for gene therapy and for sufferers of this inherited disorder. Whether this is the first gene therapy to get this close to FDA approval depends on your definition of “gene therapy.” The term can be broadly defined as treatments that use genes in some way. In theory, diseases could be cured or prevented by stopping one gene from being expressed, replacing a gene that causes a disease with one that doesn’t, or by taking a bad gene out and putting in a good gene. More precisely, Luxturna is a gene replacement therapy, Stephen Rose tells Newsweek. Rose is the chief scientific officer of the Foundation Fighting Blindness, which funded some of the preliminary research on the therapy. Keep up with this story and more by subscribing now “It’s a proof of principle for gene replacement for all sorts of other inherited rare retinal degenerations,” Rose says. Gene replacement therapy is not gene editing. The treatment does not erase the mutation that causes a disease, but it does put copies of the normal gene into cells so they can work properly. In this case, the gene therapy treats a condition called retinal dystrophy. There are actually many kinds of retinal dystrophies,...Read more
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