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Using Stem Cells to Model Diseases of the Outer Retina

Yvon, C., Ramsden, C. M., Lane, A., Powner, M. B., Da Cruz, L., Coffey, P. J. and Carr, A-J. F. (2015). Using Stem Cells to Model Diseases of the Outer Retina. Computational and Structural Biotechnology Journal, 13, pp. 382-389. doi: 10.1016/j.csbj.2015.05.001

Abstract

Retinal degeneration arises from the loss of photoreceptors or retinal pigment epithelium (RPE). It is one of the leading causes of irreversible blindness worldwide with limited effective treatment options. Generation of induced pluripotent stem cell (IPSC)-derived retinal cells and tissues from individuals with retinal degeneration is a rapidly evolving technology that holds a great potential for its use in disease modelling. IPSCs provide an ideal platform to investigate normal and pathological retinogenesis, but also deliver a valuable source of retinal cell types for drug screening and cell therapy. In this review, we will provide some examples of the ways in which IPSCs have been used to model diseases of the outer retina including retinitis pigmentosa (RP), Usher syndrome (USH), Leber congenital amaurosis (LCA), gyrate atrophy (GA), juvenile neuronal ceroid lipofuscinosis (NCL), Best vitelliform macular dystrophy (BVMD) and age related macular degeneration (AMD).

Publication Type: Article
Publisher Keywords: Disease models; Induced pluripotent stem cells; Retinitis pigmentosa; Age related macular degeneration; Leber congenital amaurosis; Inherited retinopathy
Subjects: R Medicine > RE Ophthalmology
Departments: School of Health Sciences > Optometry & Visual Science
URI: http://openaccess.city.ac.uk/id/eprint/15235
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