Gene therapy for retinal diseases: From genetics to treatment.
Journal
Indian journal of ophthalmology
ISSN: 1998-3689
Titre abrégé: Indian J Ophthalmol
Pays: India
ID NLM: 0405376
Informations de publication
Date de publication:
01 Aug 2024
01 Aug 2024
Historique:
received:
03
11
2023
accepted:
19
04
2024
medline:
30
7
2024
pubmed:
30
7
2024
entrez:
30
7
2024
Statut:
ppublish
Résumé
The gene therapy approach for retinal disorders has been considered largely over the last decade owing to the favorable outcomes of the US Food and Drug Administration-approved commercial gene therapy, Luxturna. Technological advances in recent years, such as next-generation sequencing, research in molecular pathogenesis of retinal disorders, and precise correlations with their clinical phenotypes, have contributed to the progress of gene therapies for various diseases worldwide, and more recently in India as well. Thus, considerable research is being conducted for the right choice of vectors, transgene engineering, and accessible and cost-effective large-scale vector production. Many retinal disease-specific clinical trials are presently being conducted, thereby necessitating the collation of such information as a ready reference for the scientific and clinical community. In this article, we present an overview of existing gene therapy research, which is derived from an extensive search across PubMed, Google Scholar, and clinicaltrials.gov sources. This contributes to prime the understanding of basic aspects of this cutting-edge technology and information regarding current clinical trials across many different conditions. This information will provide a comprehensive evaluation of therapies in existing use/research for personalized treatment approaches in retinal disorders.
Identifiants
pubmed: 39078952
doi: 10.4103/IJO.IJO_2902_23
pii: 02223307-202472080-00005
doi:
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
1091-1101Informations de copyright
Copyright © 2024 Copyright: © 2024 Indian Journal of Ophthalmology.
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