Newsweek

How Does Gene Therapy for Blindness Work?

The one-time treatment could restore eyesight to thousands of people.
A new gene therapy called Luxturna was being considered by the FDA on Thursday to treat retinal dystrophy, which can lead to blindness. If approved, it would be the first-ever gene replacement therapy.
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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 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.

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