Advancing therapeutic strategies for inherited retinal degeneration: Recommendations from the monaciano symposium

Debra A. Thompson, Robin R. Ali, Eyal Banin, Kari E. Branham, John G. Flannery, David M. Gamm, William W. Hauswirth, John R. Heckenlively, Alessandro Iannaccone, K. Thiran Jayasundera, Naheed W. Khan, Robert S. Molday, Mark E. Pennesi, Thomas A. Reh, Richard G. Weleber, David N. Zacks

Research output: Contribution to journalArticle

49 Scopus citations

Abstract

Although rare in the general population, retinal dystrophies occupy a central position in current efforts to develop innovative therapies for blinding diseases. This status derives, in part, from the unique biology, accessibility, and function of the retina, as well as from the synergy between molecular discoveries and transformative advances in functional assessment and retinal imaging. The combination of these factors has fueled remarkable progress in the field, while at the same time creating complex challenges for organizing collective efforts aimed at advancing translational research. The present position paper outlines recent progress in gene therapy and cell therapy for this group of disorders, and presents a set of recommendations for addressing the challenges remaining for the coming decade. It is hoped that the formulation of these recommendations will stimulate discussions among researchers, funding agencies, industry, and policy makers that will accelerate the development of safe and effective treatments for retinal dystrophies and related diseases.

Original languageEnglish (US)
Pages (from-to)918-931
Number of pages14
JournalInvestigative Ophthalmology and Visual Science
Volume56
Issue number2
DOIs
StatePublished - 2015

Keywords

  • Cell therapy
  • Disease phenotypes
  • Gene therapy
  • Outcome Measures
  • Retinal dystrophy

ASJC Scopus subject areas

  • Ophthalmology
  • Sensory Systems
  • Cellular and Molecular Neuroscience

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    Thompson, D. A., Ali, R. R., Banin, E., Branham, K. E., Flannery, J. G., Gamm, D. M., Hauswirth, W. W., Heckenlively, J. R., Iannaccone, A., Thiran Jayasundera, K., Khan, N. W., Molday, R. S., Pennesi, M. E., Reh, T. A., Weleber, R. G., & Zacks, D. N. (2015). Advancing therapeutic strategies for inherited retinal degeneration: Recommendations from the monaciano symposium. Investigative Ophthalmology and Visual Science, 56(2), 918-931. https://doi.org/10.1167/iovs.14-16049