treatment of rp

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To Treat an Inherited Retinal Disease….

In simple terms, genes are like recipes for making proteins. All the cells in our bodies “read” genetic information so they can make the critical proteins necessary to stay healthy and function properly. If there is a mistake in a gene — that is, a misspelling — a protein might not be made correctly and [...]

به وسیله |2017-10-31T10:17:43+03:30اکتبر 31st, 2017|English, دسته‌بندی نشده|بدون دیدگاه

The first gene therapy for the treatment of rp

Phase 1 studies have shown potential benefit of gene replacement in RPE65-mediated inherited retinal dystrophy. This phase 3 study assessed the efficacy and safety of voretigene neparvovec in participants whose inherited retinal dystrophy would otherwise progress to complete blindness. Methods In this open-label, randomised, controlled phase 3 trial done at two sites in the USA, [...]

به وسیله |2017-10-14T11:12:56+03:30اکتبر 14th, 2017|English, دسته‌بندی نشده|بدون دیدگاه