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Comput Struct Biotechnol J. 2015 May 6;13:382-9. doi: 10.1016/j.csbj.2015.05.001. eCollection 2015.

Using Stem Cells to Model Diseases of the Outer Retina.

Author information

1
The London Project to Cure Blindness, Division of ORBIT, Institute of Ophthalmology, University College London, 11-43 Bath Street, London EC1V 9EL, UK.
2
The London Project to Cure Blindness, Division of ORBIT, Institute of Ophthalmology, University College London, 11-43 Bath Street, London EC1V 9EL, UK ; NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust, UCL Institute of Ophthalmology, London, EC1V 2PD, UK.
3
The London Project to Cure Blindness, Division of ORBIT, Institute of Ophthalmology, University College London, 11-43 Bath Street, London EC1V 9EL, UK ; Center for Stem Cell Biology and Engineering, NRI, UC, Santa Barbara, USA.

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).

KEYWORDS:

Age related macular degeneration; Disease models; Induced pluripotent stem cells; Inherited retinopathy; Leber congenital amaurosis; Retinitis pigmentosa

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