July 2018
Volume 59, Issue 9
Open Access
ARVO Annual Meeting Abstract  |   July 2018
Optogenetics for vision restoration- translation from mice to non-human primates
Author Affiliations & Notes
  • Deniz Dalkara
    Institut de la Vision, Paris, France
    INSERM, PARIS, France
  • Footnotes
    Commercial Relationships   Deniz Dalkara, Avalanche Biotechnologies (P), Gensight Biologics (C), Gensight Biologics (F), Sanofi (F)
  • Footnotes
    Support  FFB, ERC, Gensight Biologics, Sanofi
Investigative Ophthalmology & Visual Science July 2018, Vol.59, 757. doi:
  • Views
  • Share
  • Tools
    • Alerts
      This feature is available to authenticated users only.
      Sign In or Create an Account ×
    • Get Citation

      Deniz Dalkara; Optogenetics for vision restoration- translation from mice to non-human primates. Invest. Ophthalmol. Vis. Sci. 2018;59(9):757.

      Download citation file:

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

  • Supplements

Presentation Description : Optogenetics is a biological technique, which uses light to control neurons genetically modified to express light-sensitive membrane proteins. It has become a widely used strategy in research with therapeutic applications ranging from cardiology to neurology. In terms of its clinical application, vision restoration in blind patients has come first due to the existence of an optical window to the retina and thanks to established success of gene therapy in the ocular compartment. Microbial type opsins as well as vertebrate opsins have shown promise in restoring visual responses in blind rodents and other small animals. However, it was not clear if such treatments can be translated to humans. The major challenges in translating optogenetics from mice to man are related to the lack of optimized viral vectors for targeting various populations of neurons in the primate retina and to the dose extrapolation, going from small animal eyes with minimal immune responses to a large primate eye with immune surveillance. High-level opsin expression is necessary to generate light responses in blind retinas and achieving such expression levels in a large primate eye may come at the expense of a prohibitive viral dose triggering immune responses to both the vector
and the transgene. In order to deal with these translational challenges, we studied combinations of adeno-associated virus (AAV) vectors and promoters to target optogenetic proteins to different subsets of neurons in the non-human primate retina. Our data show that successful expression of optogenetic proteins in the primate retina is cell type dependent. Furthermore, we highlight safety and efficacy of optogenetics for cell types that can be efficiently targeted with vector promoter combinations optimized for primates. Challenges remain for targeting certain neuronal populations with existing AAV technologies and for testing functional outcomes in vivo.

This is an abstract that was submitted for the 2018 ARVO Annual Meeting, held in Honolulu, Hawaii, April 29 - May 3, 2018.


This PDF is available to Subscribers Only

Sign in or purchase a subscription to access this content. ×

You must be signed into an individual account to use this feature.