期刊
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE
卷 56, 期 2, 页码 918-931出版社
ASSOC RESEARCH VISION OPHTHALMOLOGY INC
DOI: 10.1167/iovs.14-16049
关键词
retinal dystrophy; gene therapy; cell therapy; disease phenotypes; outcome measures
资金
- University of Michigan Department of Ophthalmology and Visual Sciences
- University of Tennessee Health Science Center Hamilton Eye Institute
- University of Michigan Office of the Vice President for Research
- BCM Families
- Research to Prevent Blindness
- Paul P. Lee, MD, JD
- MRC [MR/J004553/1, MR/K025589/1] Funding Source: UKRI
- Medical Research Council [MR/J004553/1, MR/K025589/1] Funding Source: researchfish
- National Institute for Health Research [NF-SI-0513-10074] Funding Source: researchfish
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.
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