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Invest Ophthalmol Vis Sci. 2015 Feb 9;56(2):918-31. doi: 10.1167/iovs.14-16049.

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

Author information

1
Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, Michigan, United States.
2
Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, Michigan, United States Division of Molecular Therapy, University College London Institute of Ophthalmology, London, England, United Kingdom.
3
Department of Ophthalmology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
4
Helen Wills Neuroscience Institute, University of California-Berkeley, Berkeley, California, United States.
5
Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, Madison, Wisconsin, United States.
6
Department of Ophthalmology, University of Florida College of Medicine, Gainesville, Florida, United States.
7
Department of Ophthalmology, Hamilton Eye Institute, University of Tennessee Health Science Center, Memphis, Tennessee, United States.
8
Department of Biochemistry and Molecular Biology, University of British Columbia, Vancouver, British Columbia, Canada.
9
Casey Eye Institute and the Department of Ophthalmology, Oregon Health and Science University, Portland, Oregon, United States.
10
Department of Biological Structure, University of Washington, Seattle, Washington, United States.
11
Casey Eye Institute and the Department of Ophthalmology, Oregon Health and Science University, Portland, Oregon, United States Department of Molecular and Medical Genetics, Oregon Health and Science University, Portland, Oregon, United States.

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.

KEYWORDS:

cell therapy; disease phenotypes; gene therapy; outcome measures; retinal dystrophy

PMID:
25667399
PMCID:
PMC4554258
DOI:
10.1167/iovs.14-16049
[Indexed for MEDLINE]
Free PMC Article

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