iPS Cells for Modelling and Treatment of Retinal Diseases

Fred K Chen; Samuel McLenachan; Michael Edel; Lyndon Da Cruz; Peter J Coffey; David A Mackey

doi:10.3390/jcm3041511

iPS Cells for Modelling and Treatment of Retinal Diseases

J Clin Med. 2014 Dec 19;3(4):1511-41. doi: 10.3390/jcm3041511.

Authors

Fred K Chen^{1

2}, Samuel McLenachan³, Michael Edel^{4

5

6

7}, Lyndon Da Cruz^{8

9}, Peter J Coffey⁹, David A Mackey¹⁰

Affiliations

¹ Centre for Ophthalmology and Visual Science (Incorporating Lions Eye Institute), The University of Western Australia, Perth WA 6009, Australia. fredchen@lei.org.au.
² Ophthalmology Department, Royal Perth Hospital, Perth WA 6009, Australia. fredchen@lei.org.au.
³ Centre for Ophthalmology and Visual Science (Incorporating Lions Eye Institute), The University of Western Australia, Perth WA 6009, Australia. SamuelMcLenachan@lei.org.au.
⁴ Control of Pluripotency Laboratory, Department of Physiological Sciences I, Faculty of Medicine, University of Barcelona, Hospital Clinic, Casanova 143, Barcelona 08036, Spain. edel.michael@gmail.com.
⁵ School of Medicine and Pharmacology, University of Western Australia, Crawley, Perth WA 6009, Australia. edel.michael@gmail.com.
⁶ Faculty of Medicine, Westmead Children's Hospital, Division of Paediatrics and Child Health, University of Sydney Medical School, Sydney NSW 2145, Australia. edel.michael@gmail.com.
⁷ Developmental and Stem Cell Biology, Victor Chang Cardiac Research Institute, Sydney NSW 2145, Australia. edel.michael@gmail.com.
⁸ Department of Vitreoretinal Surgery, Moorfields Eye Hospital, London EC1V 2PD, UK. p.coffey@ucl.ac.uk.
⁹ Division of Cellular Therapy, Institute of Ophthalmology, University College of London, London EC1V 9EL, UK. p.coffey@ucl.ac.uk.
¹⁰ Centre for Ophthalmology and Visual Science (Incorporating Lions Eye Institute), The University of Western Australia, Perth WA 6009, Australia. davidmackey@lei.org.au.

Abstract

For many decades, we have relied on immortalised retinal cell lines, histology of enucleated human eyes, animal models, clinical observation, genetic studies and human clinical trials to learn more about the pathogenesis of retinal diseases and explore treatment options. The recent availability of patient-specific induced pluripotent stem cells (iPSC) for deriving retinal lineages has added a powerful alternative tool for discovering new disease-causing mutations, studying genotype-phenotype relationships, performing therapeutics-toxicity screening and developing personalised cell therapy. This review article provides a clinical perspective on the current and potential benefits of iPSC for managing the most common blinding diseases of the eye: inherited retinal diseases and age-related macular degeneration.

Keywords: age-related macular degeneration; cell transplantation; disease modelling; genetic diagnosis; induced pluripotent; inherited retinal disease; macular dystrophy; retina; retinal dystrophy; stem cells.

Publication types

Review