Maintaining cone function in rod-cone dystrophies

JA Sahel, T Léveillard - Retinal Degenerative Diseases: Mechanisms and …, 2018 - Springer
JA Sahel, T Léveillard
Retinal Degenerative Diseases: Mechanisms and Experimental Therapy, 2018Springer
Retinal degenerative diseases are a major cause of untreatable blindness due to a loss of
photoreceptors. Recent advances in genetics and gene therapy for inherited retinal
dystrophies (IRDs) showed that therapeutic gene transfer holds a great promise for vision
restoration in people with currently incurable blinding diseases. Due to the huge genetic
heterogeneity of IRDs that represents a major obstacle for gene therapy development,
alternative therapeutic approaches are needed. This review focuses on the rescue of cone …
Abstract
Retinal degenerative diseases are a major cause of untreatable blindness due to a loss of photoreceptors. Recent advances in genetics and gene therapy for inherited retinal dystrophies (IRDs) showed that therapeutic gene transfer holds a great promise for vision restoration in people with currently incurable blinding diseases. Due to the huge genetic heterogeneity of IRDs that represents a major obstacle for gene therapy development, alternative therapeutic approaches are needed. This review focuses on the rescue of cone function as a therapeutic option for maintaining central vision in rod-cone dystrophies. It highlights recent developments in better understanding the mechanisms of action of the trophic factor RdCVF and its potential as a sight-saving therapeutic strategy.
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