Will gene therapy, stem cell therapy or low-vision devices have a greater impact on patient care for inherited retinal dystrophies in the next 5 years?

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“Inherited retinal dystrophies” is a heterogeneous group of disorders, and 5 years is a pretty short timeline. While the targeted elegance of replacing a defective gene with its normal counterpart has proven successful, the timing of treatment, the duration of effect, the mechanism of drug delivery and immunogenicity are important questions whose answers may be as heterogeneous as the diseases themselves. For RPE65 deficiency, clearly the answer is “gene therapy” as Spark Therapeutics will likely gain FDA

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