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Fibrogenesis Tissue Repair. 2015 May 15;8:9. doi: 10.1186/s13069-015-0026-9. eCollection 2015.

Prospects for clinical use of reprogrammed cells for autologous treatment of macular degeneration.

Author information

1
Control of Pluripotency Laboratory, Department of Physiological Sciences I, Faculty of Medicine, University of Barcelona, Hospital Clinic, Casanova 143, 08036 Barcelona, Spain.
2
Centre for Ophthalmology and Visual Science (Lions Eye Institute), University of Western Australia, 2 Verdun Street, Nedlands, WA 6009 Australia.
3
Moorfields Eye Hospital, 162 City Road, London, EC1V 2PD England.
4
Ear Sciences Centre, 1 Salvado Rd, Subiaco, WA 6008 Australia ; School of Surgery, University of Western Australia, 35 Stirling Highway, Nedlands, WA 6009 Australia.
5
School of Medicine and Pharmacology, University of Western Australia, 35 Stirling Highway, Nedlands, WA 6009 Australia ; PathWest, SCGH Laboratories Hospital Ave, Nedlands, WA 6009 Australia.
6
Institute for Immunology and Infectious Diseases, Murdoch University, Building 390, Discovery Way, Murdoch, Perth, WA 6150 Australia.
7
Helmholtz Zentrum München, German Research Centre for Environmental Health (GmbH), Institute of Stem Cell Research, Ingolstädter Landstraße 1, D-85764 Neuherberg, Germany.
8
Control of Pluripotency Laboratory, Department of Physiological Sciences I, Faculty of Medicine, University of Barcelona, Hospital Clinic, Casanova 143, 08036 Barcelona, Spain ; Division of Pediatrics and Child Health, Westmead Children's Hospital, Corner Hawkesbury Road and Hainsworth Street, Westmead, Sydney, NSW 2145 Australia ; School of Anatomy, Physiology & Human Biology and Centre for Cell Therapy and Regenerative Medicine (CCTRM), University of Western Australia, 35 Stirling Highway, Nedlands, WA 6009 Australia.

Abstract

Since the discovery of induced pluripotent stem cells (iPSC) in 2006, the symptoms of many human diseases have been reversed in animal models with iPSC therapy, setting the stage for future clinical development. From the animal data it is clear that iPSC are rapidly becoming the lead cell type for cell replacement therapy and for the newly developing field of iPSC-derived body organ transplantation. The first human pathology that might be treated in the near future with iPSC is age-related macular degeneration (AMD), which has recently passed the criteria set down by regulators for phase I clinical trials with allogeneic human embryonic stem cell-derived cell transplantation in humans. Given that iPSC are currently in clinical trial in Japan (RIKEN) to treat AMD, the establishment of a set of international criteria to make clinical-grade iPSC and their differentiated progeny is the next step in order to prepare for future autologous cell therapy clinical trials. Armed with clinical-grade iPSC, we can then specifically test for their threat of cancer, for proper and efficient differentiation to the correct cell type to treat human disease and then to determine their immunogenicity. Such a rigorous approach sets a far more relevant paradigm for their intended future use than non-clinical-grade iPSC. This review focuses on the latest developments regarding the first possible use of iPSC-derived retinal pigment epithelial cells in treating human disease, covers data gathered on animal models to date and methods to make clinical-grade iPSC, suggests techniques to ensure quality control and discusses possible clinical immune responses.

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