title: Gene therapies for inherited retinal disorders.
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.
authors.affiliation: Smurfit Institute of Genetics,School of Genetics and Microbiology,Trinity College Dublin,Dublin 2,Ireland.
authors.forename: G Jane
authors.initials: GJ
authors.lastname: Farrar
authors.affiliation: Smurfit Institute of Genetics,School of Genetics and Microbiology,Trinity College Dublin,Dublin 2,Ireland.
authors.forename: Sophia
authors.initials: S
authors.lastname: Millington-Ward
authors.affiliation: Smurfit Institute of Genetics,School of Genetics and Microbiology,Trinity College Dublin,Dublin 2,Ireland.
authors.forename: Naomi
authors.initials: N
authors.lastname: Chadderton
authors.affiliation: Smurfit Institute of Genetics,School of Genetics and Microbiology,Trinity College Dublin,Dublin 2,Ireland.
authors.forename: Fiona C
authors.initials: FC
authors.lastname: Mansergh
authors.affiliation: Smurfit Institute of Genetics,School of Genetics and Microbiology,Trinity College Dublin,Dublin 2,Ireland.
authors.forename: Arpad
authors.initials: A
authors.lastname: Palfi
mesh_terms: D000818:Animals; D015316:Genetic Therapy; D006801:Humans; D012164:Retinal Diseases / Q000235:genetics* / Q000628:therapy*