June 2023
Volume 64, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2023
mRNA-based therapeutics for mitochondrial optic neuropathies
Author Affiliations & Notes
  • Guy Lenaers
    MitoVasc, MitoLab, Univ Angers, Angers, France
    Service de Neurologie, CHU d'Angers, Angers, France
  • Footnotes
    Commercial Relationships   Guy Lenaers None
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science June 2023, Vol.64, 1347. doi:
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    • Get Citation

      Guy Lenaers; mRNA-based therapeutics for mitochondrial optic neuropathies. Invest. Ophthalmol. Vis. Sci. 2023;64(8):1347.

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

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Abstract

Presentation Description : Mitochondrial optic neuropathies are blinding diseases related to the loss of the retinal ganglion cells, with dominant inheritence with variants in OPA1 gene being the most frequent. To comply with OPA1 functionnal constraints, we have developped a gene therapy based on OPA1 pre-mRNA transplicing, allowing the restoration of wild-type OPA1 expression and functions, which is efficient both on haplo-insufficient and dominant negative variants. This discovery is paving the way to future clinical trials.

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

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