June 2023
Volume 64, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2023
Gene therapy to prolong cone survival
Author Affiliations & Notes
  • Connie L. Cepko
    Genetics and HHMI, Harvard Medical School, Boston, Massachusetts, United States
    Opthalmology, Harvard Medical School, Boston, Massachusetts, United States
  • Footnotes
    Commercial Relationships   Connie Cepko Biogen, Brandon Capital, Sigilon, Decibel, Code C (Consultant/Contractor), Spark Therapeutics, Code F (Financial Support)
  • Footnotes
    Support  HHMI, Spark Therapeutics
Investigative Ophthalmology & Visual Science June 2023, Vol.64, 2401. doi:
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    • Get Citation

      Connie L. Cepko; Gene therapy to prolong cone survival. Invest. Ophthalmol. Vis. Sci. 2023;64(8):2401.

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

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Abstract

Presentation Description : In many diseases where vision is lost, there is a genetic etiology. One such disease is retinitis pigmentosa (RP), where rods do not function properly, and eventually die. Night vision is thus affected in such individuals. Loss of night vision is due to the fact that rods, but not cones, express the disease gene in many forms of RP. Nonetheless, cones lose function and often die following rod death. The cones must be suffereing from problems in the environment due to the loss of the rods. Potential problems include inflammation, oxidative damage, and metabolic shortcomings. If one could address these problems, it might be possible to preserve cones and cone function in families with many different types of RP disease genes. We have created AAV vectors that express genes that can combat each of the aforementioned potential causes of cone death. They have been shown to preserve cones and cone function in 3 different models of RP in mice. This presentation will cover the results of these studies.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.

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