期刊
PROGRESS IN RETINAL AND EYE RESEARCH
卷 44, 期 -, 页码 15-35出版社
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.preteyeres.2014.10.002
关键词
Induced pluripotent stem cells; Retinal degeneration; Gene therapy; CRISPR-based genome editing; Cell transplantation
资金
- NIH [1-DP2-OD007483-01, F32 EY022834]
- NEI [EY017451]
- HHMI
- Foundation Fighting Blindness
- Stephen A. Wynn Foundation
- Grousbeck Family Foundation
- Leo, Jacques & Marion Hauser Family Vision Restoration Fund
Vision is the sense that we use to navigate the world around us. Thus it is not surprising that blindness is one of people's most feared maladies. Heritable diseases of the retina, such as age-related macular degeneration and retinitis pigmentosa, are the leading cause of blindness in the developed world, collectively affecting as many as one-third of all people over the age of 75, to some degree. For decades, scientists have dreamed of preventing vision loss or of restoring the vision of patients affected with retinal degeneration through drug therapy, gene augmentation or a cell-based transplantation approach. In this review we will discuss the use of the induced pluripotent stem cell technology to model and develop various treatment modalities for the treatment of inherited retinal degenerative disease. We will focus on the use of iPSCs for interrogation of disease pathophysiology, analysis of drug and gene therapeutics and as a source of autologous cells for cell transplantation and replacement. (C) 2014 Published by Elsevier Ltd.
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