Therapeutic landscape for inherited ocular diseases: current and emerging therapies
Inherited ocular diseases comprise a heterogeneous group of rare and complex diseases, including inherited retinal diseases (IRDs) and inherited optic neuropathies. Recent success in adeno-associated virus-based gene therapy, voretigene neparvovec (Luxturna®) for RPE65-related IRDs, has heralded rap...
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| Format: | Article |
| Language: | English |
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Wolters Kluwer – Medknow Publications
2023-01-01
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| Series: | Singapore Medical Journal |
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| Online Access: | https://journals.lww.com/10.4103/singaporemedj.SMJ-2022-179 |
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| _version_ | 1823861489981718528 |
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| author | Hwei Wuen Chan Jaslyn Oh Bart Leroy |
| author_facet | Hwei Wuen Chan Jaslyn Oh Bart Leroy |
| author_sort | Hwei Wuen Chan |
| collection | DOAJ |
| description | Inherited ocular diseases comprise a heterogeneous group of rare and complex diseases, including inherited retinal diseases (IRDs) and inherited optic neuropathies. Recent success in adeno-associated virus-based gene therapy, voretigene neparvovec (Luxturna®) for RPE65-related IRDs, has heralded rapid evolution in gene therapy platform technologies and strategies, from gene augmentation to RNA editing, as well as gene agnostic approaches such as optogenetics. This review discusses the fundamentals underlying the mode of inheritance, natural history studies and clinical trial outcomes, as well as current and emerging therapies covering gene therapy strategies, cell-based therapies and bionic vision. |
| format | Article |
| id | doaj-art-a59b389b6c0145e293de617f0d6659dc |
| institution | Kabale University |
| issn | 0037-5675 2737-5935 |
| language | English |
| publishDate | 2023-01-01 |
| publisher | Wolters Kluwer – Medknow Publications |
| record_format | Article |
| series | Singapore Medical Journal |
| spelling | doaj-art-a59b389b6c0145e293de617f0d6659dc2025-02-09T13:24:11ZengWolters Kluwer – Medknow PublicationsSingapore Medical Journal0037-56752737-59352023-01-01641172610.4103/singaporemedj.SMJ-2022-179Therapeutic landscape for inherited ocular diseases: current and emerging therapiesHwei Wuen ChanJaslyn OhBart LeroyInherited ocular diseases comprise a heterogeneous group of rare and complex diseases, including inherited retinal diseases (IRDs) and inherited optic neuropathies. Recent success in adeno-associated virus-based gene therapy, voretigene neparvovec (Luxturna®) for RPE65-related IRDs, has heralded rapid evolution in gene therapy platform technologies and strategies, from gene augmentation to RNA editing, as well as gene agnostic approaches such as optogenetics. This review discusses the fundamentals underlying the mode of inheritance, natural history studies and clinical trial outcomes, as well as current and emerging therapies covering gene therapy strategies, cell-based therapies and bionic vision.https://journals.lww.com/10.4103/singaporemedj.SMJ-2022-179antisense oligonucleotidescell replacement therapygene therapyinherited optic neuropathyinherited retinal diseases |
| spellingShingle | Hwei Wuen Chan Jaslyn Oh Bart Leroy Therapeutic landscape for inherited ocular diseases: current and emerging therapies Singapore Medical Journal antisense oligonucleotides cell replacement therapy gene therapy inherited optic neuropathy inherited retinal diseases |
| title | Therapeutic landscape for inherited ocular diseases: current and emerging therapies |
| title_full | Therapeutic landscape for inherited ocular diseases: current and emerging therapies |
| title_fullStr | Therapeutic landscape for inherited ocular diseases: current and emerging therapies |
| title_full_unstemmed | Therapeutic landscape for inherited ocular diseases: current and emerging therapies |
| title_short | Therapeutic landscape for inherited ocular diseases: current and emerging therapies |
| title_sort | therapeutic landscape for inherited ocular diseases current and emerging therapies |
| topic | antisense oligonucleotides cell replacement therapy gene therapy inherited optic neuropathy inherited retinal diseases |
| url | https://journals.lww.com/10.4103/singaporemedj.SMJ-2022-179 |
| work_keys_str_mv | AT hweiwuenchan therapeuticlandscapeforinheritedoculardiseasescurrentandemergingtherapies AT jaslynoh therapeuticlandscapeforinheritedoculardiseasescurrentandemergingtherapies AT bartleroy therapeuticlandscapeforinheritedoculardiseasescurrentandemergingtherapies |