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

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.

• Authors: 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, Monaciano Consortium
• Journal: Investigative ophthalmology & visual science (Invest Ophthalmol Vis Sci) Vol. 56 Issue 2 Pg. 918-31 (Feb 09 2015) ISSN: 1552-5783 [Electronic] United States
• PMID: 25667399 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)
• Copyright: Copyright 2015 The Association for Research in Vision and Ophthalmology, Inc.

Topics

• Congresses as Topic
• Disease Management
• Humans
• Practice Guidelines as Topic
• Retinal Degeneration (congenital, therapy)