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- W2316062018 abstract "Abstract Significant advances have been made over the last decade or two in the elucidation of the molecular pathogenesis of inherited ocular disorders. In particular, remarkable successes have been achieved in exploration of gene-based medicines for these conditions, both in preclinical and in clinical studies. Progress in the development of gene therapies targeted toward correcting the primary genetic defect or focused on modulating secondary effects associated with retinal pathologies are discussed in the review. Likewise, the recent utilization of genes encoding light-sensing molecules to provide new functions to residual retinal cells in the degenerating retina is discussed. While a great deal has been learned over the last two decades, the next decade should result in an increasing number of preclinical studies progressing to human clinical trial, an exciting prospect for patients, those active in research and development and bystanders alike." @default.
- W2316062018 created "2016-06-24" @default.
- W2316062018 creator A5041975628 @default.
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- W2316062018 creator A5055627888 @default.
- W2316062018 creator A5055993566 @default.
- W2316062018 creator A5076908022 @default.
- W2316062018 date "2014-06-20" @default.
- W2316062018 modified "2023-09-24" @default.
- W2316062018 title "Gene therapies for inherited retinal disorders" @default.
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