title: [Gene Therapy for Inherited RETINAL AND OPTIC NERVE Disorders: Current Knowledge].
abstract: The aim of this review is to provide a comprehensive summary of current gene therapy clinical trials for monogenic and optic nerve disorders.The number of genes for which gene-based therapies are being developed is growing. At the time of writing this review gene-based clinical trials have been registered for Leber congenital amaurosis 2 (LCA2), retinitis pigmentosa 38, Usher syndrome 1B, Stargardt disease, choroideremia, achromatopsia, Leber hereditary optic neuropathy (LHON) and X-linked retinoschisis. Apart from RPE65 gene therapy for LCA2 and MT-ND4 for LHON which has reached phase III, all other trials are in investigation phase I and II, i.e. testing the efficacy and safety.Because of the relatively easy accessibility of the retina and its ease of visualization which allows monitoring of efficacy, gene-based therapies for inherited retinal disorders represent a very promising treatment option. With the development of novel therapeutic approaches, the importance of establishing not only clinical but also molecular genetic diagnosis is obvious.Key words: gene therapy, monogenic retinal diseases, optic nerve atrophy, mitochondrial disease.
authors.forename: Ľ
authors.initials: Ľ
authors.lastname: Ďuďáková
authors.forename: B
authors.initials: B
authors.lastname: Kousal
authors.forename: H
authors.initials: H
authors.lastname: Kolářová
authors.forename: L
authors.initials: L
authors.lastname: Hlavatá
authors.forename: P
authors.initials: P
authors.lastname: Lišková
mesh_terms: D015785:Eye Diseases, Hereditary / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D009901:Optic Nerve Diseases / Q000235:genetics / Q000628:therapy*; D012164:Retinal Diseases / Q000235:genetics / Q000628:therapy*