Antisense-mediated splicing correction approaches for retinal dystrophies and dysfunctions
Published: Jun 18, 2015
Abstract
Retinal dystrophies are a large and extremely variegate group of diseases causing visual impairments and eventually blindness. As of today, no cures are available for such conditions, and the most advanced clinical approaches null are based on classical gene transfer techniques. The recent development of genetic tools designed to manipulate splicing offers a unique opportunity to target several of these diseases, potentially reaching a highly...
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