Table of Contents
Genes, Volume 10, Issue 9 (September 2019) – 99 articles
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Cover Story (view full-size image) Long considered incurable, inherited retinal diseases (IRDs) are currently at the forefront of the [...] Read more. Long considered incurable, inherited retinal diseases (IRDs) are currently at the forefront of the development of novel therapeutic strategies. Many of these strategies focus on resolving the primary cause of the disease, a mutation in one of the many genes associated with IRD. This group of therapeutic strategies, coined ‘molecular therapies’ can act at the DNA, RNA or protein level, and all aim to improve visual function, or at least slow down or halt disease progression, following administration to the retina. Which of the strategies will be most useful for each patient depends on many factors, including the type of genetic defect, its prevalence, target gene and cell type, delivery methods, and the state of the disease at the possible time of intervention. The different approaches, as well as the many factors that need to be taken into account, are summarized in this review. View this paper.