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    Updates on Gene Therapy for Diabetic Retinopathy

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    Author
    Wang, J-H; Roberts, GE; Liu, G-S
    Date
    2020-05-16
    Source Title
    Current Diabetes Reports
    Publisher
    CURRENT MEDICINE GROUP
    University of Melbourne Author/s
    Liu, Guei-Sheung; Wang, Jiang Hui
    Affiliation
    Ophthalmology (Eye & Ear Hospital)
    Metadata
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    Document Type
    Journal Article
    Citations
    Wang, J. -H., Roberts, G. E. & Liu, G. -S. (2020). Updates on Gene Therapy for Diabetic Retinopathy. CURRENT DIABETES REPORTS, 20 (7), https://doi.org/10.1007/s11892-020-01308-w.
    Access Status
    Access this item via the Open Access location
    URI
    http://hdl.handle.net/11343/254438
    DOI
    10.1007/s11892-020-01308-w
    Open Access URL
    https://europepmc.org/articles/PMC7228867?pdf=render
    Abstract
    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. 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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