July 2019
Volume 60, Issue 9
Open Access
ARVO Annual Meeting Abstract  |   July 2019
Gene delivery to the trabecular meshwork for target validation, development of disease models, and the treatment of glaucoma
Author Affiliations & Notes
  • Abbot F Clark
    Cell Biology & Anatomy, University of North Texas HSC, Fort Worth, Texas, United States
  • Footnotes
    Commercial Relationships   Abbot Clark, Goodmans, LLP (C), Lung Therapeutics Inc (F), Unity Biotechnology (F)
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science July 2019, Vol.60, 1038. doi:
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      Abbot F Clark; Gene delivery to the trabecular meshwork for target validation, development of disease models, and the treatment of glaucoma . Invest. Ophthalmol. Vis. Sci. 2019;60(9):1038.

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

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Abstract

Presentation Description : Glaucomatous damage to the trabecular meshwork (TM) is responsible for the development ocular hypertension in glaucoma. Adenovirus (Ad5) and adeno-associated virus (scAAV2) vectors have selective tropism for the TM of human, non-human primate, rat, and mouse eyes. A number of aberrant signaling pathways (WNT, BMP, CTGF, CD44, GRb, COCH, etc.) have been implicated in glaucomatous damage to the TM, and viral vectors have been used to validate the effects of these pathways to generate ocular hypertension in ex vivo perfusion cultured anterior segments as well as in rodent eyes. Long-term expression of these transgenes after TM transduction has led to the development of rodent glaucoma models that more effectively mimic pathological damage to the glaucomatous TM and development of ocular hypertension. Several gene therapy approaches have been used to inhibit glucocorticoid-induced ocular hypertension and glaucoma, and CRISPR/Cas9 gene editing has been used to reduce IOP in a mouse model of MYOC glaucoma. Gene delivery and gene editing of the TM are potential new disease modifying therapies for the treatment of glaucoma by directly addressing the molecular pathogenic processes in the TM.

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

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