Journal article
Gene therapy using stem cells
Cold Spring Harbor perspectives in medicine, Vol.5(4), a017434
11/13/2014
DOI: 10.1101/cshperspect.a017434
PMCID: PMC4382729
PMID: 25395375
Abstract
Viral-mediated gene augmentation therapy has recently shown success in restoring vision to patients with retinal degenerative disorders. Key to this success was the availability of animal models that accurately presented the human phenotype to test preclinical efficacy and safety. These exciting studies support the use of gene therapy in the treatment of devastating retinal degenerative diseases. In some cases, however, in vivo gene therapy for retinal degeneration would not be effective because the cell types targeted are no longer present. The development of somatic cell reprogramming methods provides an attractive source of autologous cells for transplantation and treatment of retinal degenerative disease. This article explores the development of gene therapy and patient-derived stem cells for the purpose of restoring vision to individuals suffering from inherited retinal degenerations.
Details
- Title: Subtitle
- Gene therapy using stem cells
- Creators
- Erin R Burnight - University of IowaLuke A Wiley - University of IowaRobert F Mullins - University of IowaEdwin M Stone - University of IowaBudd A Tucker - University of Iowa
- Resource Type
- Journal article
- Publication Details
- Cold Spring Harbor perspectives in medicine, Vol.5(4), a017434
- DOI
- 10.1101/cshperspect.a017434
- PMID
- 25395375
- PMCID
- PMC4382729
- 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
- 11/13/2014
- Academic Unit
- Iowa Neuroscience Institute; Ophthalmology and Visual Sciences
- Record Identifier
- 9983979996402771
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