Journal article
Concise Review: Patient-Specific Stem Cells to Interrogate Inherited Eye Disease
Stem cells translational medicine, Vol.5(2), pp.132-140
02/2016
DOI: 10.5966/sctm.2015-0206
PMCID: PMC4729558
PMID: 26683869
Abstract
Whether we are driving to work or spending time with loved ones, we depend on our sense of vision to interact with the world around us. Therefore, it is understandable why blindness for many is feared above death itself. Heritable diseases of the retina, such as glaucoma, age-related macular degeneration, and retinitis pigmentosa, are major causes of blindness worldwide. The recent success of gene augmentation trials for the treatment of RPE65-associated Leber congenital amaurosis has underscored the need for model systems that accurately recapitulate disease. With the advent of patient-specific induced pluripotent stem cells (iPSCs), researchers are now able to obtain disease-specific cell types that would otherwise be unavailable for molecular analysis. In the present review, we discuss how the iPSC technology is being used to confirm the pathogenesis of novel genetic variants, interrogate the pathophysiology of disease, and accelerate the development of patient-centered treatments. Significance: Stem cell technology has created the opportunity to advance treatments for multiple forms of blindness. Researchers are now able to use a person's cells to generate tissues found in the eye. This technology can be used to elucidate the genetic causes of disease and develop treatment strategies. In the present review, how stem cell technology is being used to interrogate the pathophysiology of eye disease and accelerate the development of patient-centered treatments is discussed.
Details
- Title: Subtitle
- Concise Review: Patient-Specific Stem Cells to Interrogate Inherited Eye Disease
- Creators
- Joseph C Giacalone - Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USALuke A Wiley - Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USAErin R Burnight - Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USAAllison E Songstad - Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USARobert F Mullins - Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USAEdwin M Stone - Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA Howard Hughes Medical Institute, University of Iowa, Iowa City, Iowa, USABudd A Tucker - Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA budd-tucker@uiowa.edu
- Resource Type
- Journal article
- Publication Details
- Stem cells translational medicine, Vol.5(2), pp.132-140
- Publisher
- United States
- DOI
- 10.5966/sctm.2015-0206
- PMID
- 26683869
- PMCID
- PMC4729558
- ISSN
- 2157-6564
- eISSN
- 2157-6580
- Grant note
- R01 EY024605 / NEI NIH HHS DP2 OD007483 / NIH HHS 1-DP2-OD007483-01 / NIH HHS Howard Hughes Medical Institute T32 GM007337 / NIGMS NIH HHS EY024605 / NEI NIH HHS F32 EY022834 / NEI NIH HHS
- Language
- English
- Date published
- 02/2016
- Academic Unit
- The University of Iowa Institute for Vision Research; Iowa Neuroscience Institute; John and Marcia Carver Nonprofit Genetic Testing Laboratory; Ophthalmology and Visual Sciences
- Record Identifier
- 9984070313802771
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