title: 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: Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, Michigan, United States.
authors: Debra A
authors: DA
authors: Thompson
authors: 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.
authors: Robin R
authors: RR
authors: Ali
authors: Department of Ophthalmology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
authors: Eyal
authors: E
authors: Banin
authors: Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, Michigan, United States.
authors: Kari E
authors: KE
authors: Branham
authors: Helen Wills Neuroscience Institute, University of California-Berkeley, Berkeley, California, United States.
authors: John G
authors: JG
authors: Flannery
authors: Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, Madison, Wisconsin, United States.
authors: David M
authors: DM
authors: Gamm
authors: Department of Ophthalmology, University of Florida College of Medicine, Gainesville, Florida, United States.
authors: William W
authors: WW
authors: Hauswirth
authors: Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, Michigan, United States.
authors: John R
authors: JR
authors: Heckenlively
authors: Department of Ophthalmology, Hamilton Eye Institute, University of Tennessee Health Science Center, Memphis, Tennessee, United States.
authors: Alessandro
authors: A
authors: Iannaccone
authors: Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, Michigan, United States.
authors: K Thiran
authors: KT
authors: Jayasundera
authors: Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, Michigan, United States.
authors: Naheed W
authors: NW
authors: Khan
authors: Department of Biochemistry and Molecular Biology, University of British Columbia, Vancouver, British Columbia, Canada.
authors: Robert S
authors: RS
authors: Molday
authors: Casey Eye Institute and the Department of Ophthalmology, Oregon Health and Science University, Portland, Oregon, United States.
authors: Mark E
authors: ME
authors: Pennesi
authors: Department of Biological Structure, University of Washington, Seattle, Washington, United States.
authors: Thomas A
authors: TA
authors: Reh
authors: 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.
authors: Richard G
authors: RG
authors: Weleber
authors: Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, Michigan, United States.
authors: David N
authors: DN
authors: Zacks
mesh_terms: D003226:Congresses as Topic; D019468:Disease Management; D006801:Humans; D017410:Practice Guidelines as Topic; D012162:Retinal Degeneration / Q000151:congenital* / Q000628:therapy*
keywords: cell therapy; disease phenotypes; gene therapy; outcome measures; retinal dystrophy