Therapeutic landscape for inherited ocular diseases: Current and emerging therapies
Singapore medical journal
;
: 17-26, 2023.
Article
Dans Anglais
| WPRIM
| ID: wpr-969661
ABSTRACT
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.
Texte intégral:
Disponible
Indice:
WPRIM (Pacifique occidental)
Sujet Principal:
Maladies de l'oeil
Limites du sujet:
Humains
langue:
Anglais
Texte intégral:
Singapore medical journal
Année:
2023
Type:
Article
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