Journal article
Stem cells as tools for studying the genetics of inherited retinal degenerations
Cold Spring Harbor perspectives in medicine, Vol.5(5), pp.a017160-a017160
12/11/2014
DOI: 10.1101/cshperspect.a017160
PMCID: PMC4448588
PMID: 25502747
Abstract
The ability to provide early clinical intervention for inherited disorders is heavily dependent on knowledge of a patient's disease-causing mutations and the resultant pathophysiologic mechanism(s). Without knowing a patient's disease-causing gene, and how gene mutations alter the health and functionality of affected cells, it would be difficult to develop and deliver patient-specific molecular or small molecule therapies. Many believe that the field of stem cell biology holds the keys to the future development of disease-, patient-, and cell-specific therapies. In the case of the eye, which is susceptible to an extremely common late-onset degenerative disease known as age-related macular degeneration, stem cell-based therapies could increase the quality of life for millions of patients worldwide. Furthermore, autologous, patient-specific induced pluripotent stem cells could be a viable source to treat rare Mendelian retinal degenerative diseases such as retinitis pigmentosa, Stargardt disease, and Best disease, to name a few.
Details
- Title: Subtitle
- Stem cells as tools for studying the genetics of inherited retinal degenerations
- Creators
- Luke A Wiley - Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, Iowa 52242Erin R Burnight - Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, Iowa 52242Robert F Mullins - Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, Iowa 52242Edwin M Stone - Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, Iowa 52242 Howard Hughes Medical Institute, University of Iowa, Iowa City, Iowa 52242Budd A Tucker - Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, Iowa 52242
- Resource Type
- Journal article
- Publication Details
- Cold Spring Harbor perspectives in medicine, Vol.5(5), pp.a017160-a017160
- DOI
- 10.1101/cshperspect.a017160
- PMID
- 25502747
- PMCID
- PMC4448588
- NLM abbreviation
- Cold Spring Harb Perspect Med
- ISSN
- 2157-1422
- eISSN
- 2157-1422
- Publisher
- United States
- Grant note
- EY017451 / NEI NIH HHS R01 EY017451 / NEI NIH HHS DP2 OD007483 / NIH HHS 1-DP2-OD007483-01 / NIH HHS Howard Hughes Medical Institute F32 EY022834 / NEI NIH HHS
- Language
- English
- Date published
- 12/11/2014
- Academic Unit
- Iowa Neuroscience Institute; Ophthalmology and Visual Sciences
- Record Identifier
- 9983979973702771
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