July 2019
Volume 60, Issue 9
Open Access
ARVO Annual Meeting Abstract  |   July 2019
Overview of Gene Therapy in Anterior Segment
Author Affiliations & Notes
  • Alexander V Ljubimov
    Regenerative Medicine Institute, Cedars-Sinai Medical Center, West Hollywood, California, United States
    Medicine, UCLA School of Medicine, Los Angeles, California, United States
  • Footnotes
    Commercial Relationships   Alexander Ljubimov, Arrogene, Inc. (I)
  • Footnotes
    Support  EY13431
Investigative Ophthalmology & Visual Science July 2019, Vol.60, 1037. doi:
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      Alexander V Ljubimov; Overview of Gene Therapy in Anterior Segment. Invest. Ophthalmol. Vis. Sci. 2019;60(9):1037.

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      © ARVO (1962-2015); The Authors (2016-present)

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Presentation Description : The accessibility and a variety of monitoring options make gene therapy an easy option for the anterior segment diseases affecting cornea, conjunctiva, trabecular meshwork, and lacrimal gland. Although there are still no clinical trials to date, gene therapy has been successfully tested in model systems and in animals using various viruses as delivery vehicles, as well as plasmids, microRNA and nanoconstructs that were administered topically and by intrastromal, subconjunctival or anterior chamber injections. Successful use in the cornea included amelioration of mucopolysaccharidosis type VII (β-glucuronidase gene delivery), scarring, wound healing and burn injury (decorin, Smad7, BMP7, c-met genes; antisense to opioid growth factor receptor gene, inhibitor of miR-146a), graft survival (interleukin genes, bcl-xL gene), herpes keratitis (HSV glycoprotein genes), and pathologic neovascularization (s-Flt1, Flt23K, PEDF, endostatin genes, and miR-204). Conjunctival scarring was counteracted by gene therapy to increase the expression of Smad7, PPARγ, and dominant negative p38 MAPkinase. In the lacrimal gland, dry eye symptoms were alleviated by injection of TNF-α inhibitor, and IL-10 injection improved signs of Sjögren’s syndrome. Nanoparticle delivery of MUC5AC gene also increased tear production in dry eye. In the trabecular meshwork/outflow system, gene therapy with cyclooxygenase-2, prostaglandin F synthase or matrix metalloproteinase-3 upregulation increased outflow facility and decreased IOP. Overall, gene therapy using various vectors and molecular targets has been successful against various conditions affecting the anterior segment. These results hold promise for future clinical applications. The current trends include the prevalence of topical gene administration and increasing use of nano carriers for gene delivery.

This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.


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