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Maintaining Cone Function in Rod-Cone Dystrophies

  • José-Alain Sahel
  • Thierry Léveillard
Conference paper
Part of the Advances in Experimental Medicine and Biology book series (AEMB, volume 1074)

Abstract

Retinal degenerative diseases are a major cause of untreatable blindness due to a loss of photoreceptors. Recent advances in genetics and gene therapy for inherited retinal dystrophies (IRDs) showed that therapeutic gene transfer holds a great promise for vision restoration in people with currently incurable blinding diseases. Due to the huge genetic heterogeneity of IRDs that represents a major obstacle for gene therapy development, alternative therapeutic approaches are needed. This review focuses on the rescue of cone function as a therapeutic option for maintaining central vision in rod-cone dystrophies. It highlights recent developments in better understanding the mechanisms of action of the trophic factor RdCVF and its potential as a sight-saving therapeutic strategy.

Keywords

Retinal degeneration Photoreceptors Rod-derived cone viability factor Aerobic glycolysis Neuroprotective gene therapy Nucleoredoxin-like-1 

Notes

Financial Support

LABEX LIFESENSES [ANR-10-LABX-65], Foundation Fighting Blindness.

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Copyright information

© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  • José-Alain Sahel
    • 1
    • 2
    • 3
  • Thierry Léveillard
    • 1
  1. 1.Sorbonne Universités, UPMC Univ Paris 06, INSERM U968, CNRS UMR 7210, Institut de la VisionParisFrance
  2. 2.CHNO des Quinze-Vingts, DHU Sight Restore, INSERM-DGOS CIC 1423ParisFrance
  3. 3.Department of OphthalmologyThe University of Pittsburgh School of MedicinePittsburghUSA

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