New gene therapy gives hope to people born with inherited eye disorder
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Sten Andreasson, Lund University
The first successful gene therapy for an inherited form of blindness was recently reviewed by an expert panel of advisers at US Food and Drug Administration (FDA). They unanimously voted in recommendation of the treatment. The FDA now has until January 12, 2018 to approve the treatment.
Spark Therapeutics, an American biotech firm, developed the therapy (Luxturna) to treat Leber congenital amaurosis (LCA), an inherited eye disorder. Although LCA is very rare (affecting about one in 80,000 people) it is the most common cause of inherited sight loss in young people.
The disease causes cells in the eye, known as the retinal pigment epithelium, to stop working properly. This thin layer of cells supports and nourishes the retina, the light-sensitive tissue at the back of the eye.
LCA symptoms, such as night blindness, roving eyes (nystagmus) and tunnel vision, start in early childhood and usually progress to complete sight loss b...
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