Over the past 3 decades, global research efforts have unveiled the genetic complexity of the group of rare disorders collectively referred to as retinal dystrophies. Causative mutations for dozens of retinal dystrophies have now been identified, resulting in the discovery of multiple new genes, biological pathways necessary for photoreceptor cell function, and pathogenetic mechanisms involved in retinal degeneration.
The paper outlines recent progress in gene therapy for this group of disorders, and presents a set of recommendations for addressing the challenges remaining for the coming decade. It is hoped that the formulation of these recommendations will stimulate discussions among researchers, funding agencies, industry, and policy makers that will accelerate the development of safe and effective treatments for retinal dystrophies and related diseases.
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