Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium.


Journal Article (Review)

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.

Full Text

Duke Authors

Cited Authors

  • Thompson, DA; Ali, RR; Banin, E; Branham, KE; Flannery, JG; Gamm, DM; Hauswirth, WW; Heckenlively, JR; Iannaccone, A; Jayasundera, KT; Khan, NW; Molday, RS; Pennesi, ME; Reh, TA; Weleber, RG; Zacks, DN; Monaciano Consortium,

Published Date

  • February 9, 2015

Published In

Volume / Issue

  • 56 / 2

Start / End Page

  • 918 - 931

PubMed ID

  • 25667399

Pubmed Central ID

  • 25667399

Electronic International Standard Serial Number (EISSN)

  • 1552-5783

International Standard Serial Number (ISSN)

  • 0146-0404

Digital Object Identifier (DOI)

  • 10.1167/iovs.14-16049


  • eng