Inherited retinal diseases: Therapeutics, clinical trials and end points-A review.
gene therapy
inherited retinal disease
pharmacological therapy
retina
stem cell
Journal
Clinical & experimental ophthalmology
ISSN: 1442-9071
Titre abrégé: Clin Exp Ophthalmol
Pays: Australia
ID NLM: 100896531
Informations de publication
Date de publication:
Apr 2021
Apr 2021
Historique:
revised:
22
02
2021
received:
19
11
2020
accepted:
01
03
2021
pubmed:
10
3
2021
medline:
1
9
2021
entrez:
9
3
2021
Statut:
ppublish
Résumé
Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous group of disorders characterised by photoreceptor degeneration or dysfunction. These disorders typically present with severe vision loss that can be progressive, with disease onset ranging from congenital to late adulthood. The advances in genetics, retinal imaging and molecular biology, have conspired to create the ideal environment for establishing treatments for IRDs, with the first approved gene therapy and the commencement of multiple clinical trials. The scope of this review is to familiarise clinicians and scientists with the current management and the prospects for novel therapies for: (1) macular dystrophies, (2) cone and cone-rod dystrophies, (3) cone dysfunction syndromes, (4) Leber congenital amaurosis, (5) rod-cone dystrophies, (6) rod dysfunction syndromes and (7) chorioretinal dystrophies. We also briefly summarise the investigated end points for the ongoing trials.
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
270-288Subventions
Organisme : Foundation Fighting Blindness (USA)
Organisme : Retina UK
Organisme : Moorfields Eye Charity
Organisme : Moorfields Eye Hospital Special Trustees
Organisme : Leventis Foundation
Organisme : Onassis Foundation
Organisme : Fight for Sight (UK)
Organisme : Macular Society (UK)
Organisme : National Institute for Health Research Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology
Organisme : Wellcome Trust
ID : 099173/Z/12/Z
Pays : United Kingdom
Informations de copyright
© 2021 The Authors. Clinical & Experimental Ophthalmology published by John Wiley & Sons Australia, Ltd on behalf of Royal Australian and New Zealand College of Ophthalmologists.
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