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Updates on Gene Therapy for Diabetic Retinopathy

PURPOSE OF REVIEW: Diabetic retinopathy (DR), a leading cause of visual impairment in the developed country, is characterized by vascular lesions and neuronal damage of the retina. Treatment options for this condition are currently limited. The advent of therapy targeting vascular endothelial growth...

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Autores principales: Wang, Jiang-Hui, Roberts, Georgina Eloise, Liu, Guei-Sheung
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer US 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7228867/
https://www.ncbi.nlm.nih.gov/pubmed/32415508
http://dx.doi.org/10.1007/s11892-020-01308-w
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author Wang, Jiang-Hui
Roberts, Georgina Eloise
Liu, Guei-Sheung
author_facet Wang, Jiang-Hui
Roberts, Georgina Eloise
Liu, Guei-Sheung
author_sort Wang, Jiang-Hui
collection PubMed
description PURPOSE OF REVIEW: Diabetic retinopathy (DR), a leading cause of visual impairment in the developed country, is characterized by vascular lesions and neuronal damage of the retina. Treatment options for this condition are currently limited. The advent of therapy targeting vascular endothelial growth factor (VEGF) demonstrated significant benefits to patients with DR. However, this treatment is limited by its short half-life and requirement for frequent invasive intravitreal injections. In addition, many patients failed to achieve clinically significant improvement in visual function. Gene therapy has the potential to provide an alternative treatment for DR with distinct advantages, such as longer therapeutic effect, less injection frequency, ability to intervene at disease onset, and potentially fewer side effects. RECENT FINDINGS: Strategies for gene therapy in DR, stemming from the current understanding of the disease pathogenesis, focus on the inhibition of neovascularization and protection of neurovascular degeneration in the retina. Studies with promising results have mainly focussed on animal models due to efficacy and safety concerns, despite a number of successful preclinical studies using adeno-associated virus-mediated transduction to treat both vascular dysfunction and neuronal degeneration. With the optimization of delivery vectors, transgene regulation, and outcome measure, gene therapy will potentially become available for patients with DR. SUMMARY: This review provides an update on the current strategies utilized in DR gene therapy research. Several barriers to the clinical application of gene therapy for DR are highlighted, and future directions for this research are proposed.
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spelling pubmed-72288672020-05-18 Updates on Gene Therapy for Diabetic Retinopathy Wang, Jiang-Hui Roberts, Georgina Eloise Liu, Guei-Sheung Curr Diab Rep Immunology, Transplantation, and Regenerative Medicine (L Piemonti and V Sordi, Section Editors) PURPOSE OF REVIEW: Diabetic retinopathy (DR), a leading cause of visual impairment in the developed country, is characterized by vascular lesions and neuronal damage of the retina. Treatment options for this condition are currently limited. The advent of therapy targeting vascular endothelial growth factor (VEGF) demonstrated significant benefits to patients with DR. However, this treatment is limited by its short half-life and requirement for frequent invasive intravitreal injections. In addition, many patients failed to achieve clinically significant improvement in visual function. Gene therapy has the potential to provide an alternative treatment for DR with distinct advantages, such as longer therapeutic effect, less injection frequency, ability to intervene at disease onset, and potentially fewer side effects. RECENT FINDINGS: Strategies for gene therapy in DR, stemming from the current understanding of the disease pathogenesis, focus on the inhibition of neovascularization and protection of neurovascular degeneration in the retina. Studies with promising results have mainly focussed on animal models due to efficacy and safety concerns, despite a number of successful preclinical studies using adeno-associated virus-mediated transduction to treat both vascular dysfunction and neuronal degeneration. With the optimization of delivery vectors, transgene regulation, and outcome measure, gene therapy will potentially become available for patients with DR. SUMMARY: This review provides an update on the current strategies utilized in DR gene therapy research. Several barriers to the clinical application of gene therapy for DR are highlighted, and future directions for this research are proposed. Springer US 2020-05-16 2020 /pmc/articles/PMC7228867/ /pubmed/32415508 http://dx.doi.org/10.1007/s11892-020-01308-w Text en © Springer Science+Business Media, LLC, part of Springer Nature 2020 This article is made available via the PMC Open Access Subset for unrestricted research re-use and secondary analysis in any form or by any means with acknowledgement of the original source. These permissions are granted for the duration of the World Health Organization (WHO) declaration of COVID-19 as a global pandemic.
spellingShingle Immunology, Transplantation, and Regenerative Medicine (L Piemonti and V Sordi, Section Editors)
Wang, Jiang-Hui
Roberts, Georgina Eloise
Liu, Guei-Sheung
Updates on Gene Therapy for Diabetic Retinopathy
title Updates on Gene Therapy for Diabetic Retinopathy
title_full Updates on Gene Therapy for Diabetic Retinopathy
title_fullStr Updates on Gene Therapy for Diabetic Retinopathy
title_full_unstemmed Updates on Gene Therapy for Diabetic Retinopathy
title_short Updates on Gene Therapy for Diabetic Retinopathy
title_sort updates on gene therapy for diabetic retinopathy
topic Immunology, Transplantation, and Regenerative Medicine (L Piemonti and V Sordi, Section Editors)
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7228867/
https://www.ncbi.nlm.nih.gov/pubmed/32415508
http://dx.doi.org/10.1007/s11892-020-01308-w
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