The clinical success of gene replacement therapies in recent years has served as a proof of concept for the treatment of inherited retinal degenerations using adeno-associated virus (AAV) as viral vector. However, inherited retinal degenerative diseases showcase a broad genetic and mechanistic heterogeneity, challenging the development of mutation-specific therapies for each specific mutation. Mutation-independent approaches must be developed to slow down retinal degeneration regardless of the underlying genetic mutation and onset of the disease. New understanding of cell death mechanisms in rod-cone dystrophies have led to promising rescue of photoreceptor cell death by virally mediating expression of anti-apoptotic factors and secretion of retinal neurotrophic factors. Optogenetic therapies are also able to restore light sensitivities in blind retinas.
CITATION STYLE
Fortuny, C., & Flannery, J. G. (2018). Mutation-independent gene therapies for rod-cone dystrophies. In Advances in Experimental Medicine and Biology (Vol. 1074, pp. 75–81). Springer New York LLC. https://doi.org/10.1007/978-3-319-75402-4_10
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