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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

FDA Unanimously Recommends Approval for Spark’s RPE65

FDA Committee Unanimously Recommends Approval for Spark’s RPE65 Gene Therapy – Final Decision Due in January 2018 Spark Therapeutics has taken a major step closer to gaining marketing approval for its vision-restoring gene therapy for people with RPE65 mutations causing Leber congenital amaurosis (LCA) and retinitis pigmentosa. At the conclusion of a public hearing on [...]

به وسیله |2017-10-18T12:47:44+03:30اکتبر 18th, 2017|English, دسته‌بندی نشده|بدون دیدگاه