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Dysregulation of cGMP is a pathological hallmark of inherited retinal degenerations (RD) affecting photoreceptors, the sensory cells of the retina. These RDs, including Retinitis Pigmentosa, Lebers Congenital Amaurosis, and Achromatopsia, are major causes of blindness, affecting approximately one in every 2000 individuals worldwide, and remain without effective treatment. In photoreceptors, cGMP i ...

"For many disabling or fatal diseases, there is pre-clinical or clinical evidence of the potential therapeutic efficacy of gene therapy and, yet, the limitations of current gene transfer technologies have prevented success or even caused serious adverse events leading to termination of trials. PERSIST will explore the use of highly innovative gene-modifying and delivery technologies and capitalize ...