Therapeutic landscape for inherited ocular diseases: Current and emerging therapies.
10.4103/singaporemedj.SMJ-2022-179
- Author:
Hwei Wuen CHAN
1
,
2
;
Jaslyn OH
3
;
Bart LEROY
4
,
5
,
6
,
7
Author Information
1. Department of Ophthalmology, National University Hospital
2. Department of Ophthalmology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore.
3. Department of Ophthalmology, National University Hospital, Singapore.
4. Department of Ophthalmology, Ghent University Hospital
5. Department of Head and Skin, Ghent University
6. Center for Medical Genetics, Ghent University Hospital, Ghent, Belgium
7. Division of Ophthalmology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
- Publication Type:Review
- Keywords:
Antisense oligonucleotides;
cell replacement therapy;
gene therapy;
inherited optic neuropathy;
inherited retinal diseases
- MeSH:
Humans;
Eye Diseases/therapy*
- From:Singapore medical journal
2023;64(1):17-26
- CountrySingapore
- Language:English
-
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.
