Gene Therapy Intervention in Neovascular Eye Disease: A Recent Update.
Animals
CRISPR-Cas Systems
Clinical Trials as Topic
Disease Management
Disease Susceptibility
Eye Diseases
/ etiology
Gene Editing
Genetic Therapy
/ methods
Humans
Neovascularization, Pathologic
/ genetics
Platelet-Derived Growth Factor
/ genetics
Treatment Outcome
Vascular Endothelial Growth Factor A
/ genetics
VEGF
age-related macular degeneration
diabetic retinopathy
eye
gene therapy
neovascularization
Journal
Molecular therapy : the journal of the American Society of Gene Therapy
ISSN: 1525-0024
Titre abrégé: Mol Ther
Pays: United States
ID NLM: 100890581
Informations de publication
Date de publication:
07 10 2020
07 10 2020
Historique:
received:
28
04
2020
revised:
15
06
2020
accepted:
26
06
2020
pubmed:
11
7
2020
medline:
12
8
2021
entrez:
11
7
2020
Statut:
ppublish
Résumé
Aberrant growth of blood vessels (neovascularization) is a key feature of severe eye diseases that can cause legal blindness, including neovascular age-related macular degeneration (nAMD) and diabetic retinopathy (DR). The development of anti-vascular endothelial growth factor (VEGF) agents has revolutionized the treatment of ocular neovascularization. Novel proangiogenic targets, such as angiopoietin and platelet-derived growth factor (PDGF), are under development for patients who respond poorly to anti-VEGF therapy and to reduce adverse effects from long-term VEGF inhibition. A rapidly advancing area is gene therapy, which may provide significant therapeutic benefits. Viral vector-mediated transgene delivery provides the potential for continuous production of antiangiogenic proteins, which would avoid the need for repeated anti-VEGF injections. Gene silencing with RNA interference to target ocular angiogenesis has been investigated in clinical trials. Proof-of-concept gene therapy studies using gene-editing tools such as CRISPR-Cas have already been shown to be effective in suppressing neovascularization in animal models, highlighting the therapeutic potential of the system for treatment of aberrant ocular angiogenesis. This review provides updates on the development of anti-VEGF agents and novel antiangiogenic targets. We also summarize current gene therapy strategies already in clinical trials and those with the latest approaches utilizing CRISPR-Cas gene editing against aberrant ocular neovascularization.
Identifiants
pubmed: 32649860
pii: S1525-0016(20)30341-5
doi: 10.1016/j.ymthe.2020.06.029
pmc: PMC7544979
pii:
doi:
Substances chimiques
Platelet-Derived Growth Factor
0
Vascular Endothelial Growth Factor A
0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
2120-2138Informations de copyright
Copyright © 2020 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved.
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