title: A strategy to ensure safety of stem cell-derived retinal pigment epithelium cells.
abstract: Cell replacement and regenerative therapy using embryonic stem cell-derived material holds promise for the treatment of several pathologies. However, the safety of this approach is of prime importance given the teratogenic potential of residual stem cells, if present in the differentiated cell product. Using the example of embryonic stem cell-derived retinal pigment epithelium (RPE) for the treatment of age-related macular degeneration, we present a novel strategy for ensuring the absence of stem cells in the RPE population. Based on an unbiased screening approach, we identify and validate the expression of CD59, a cell surface marker expressed on RPE but absent on stem cells. We further demonstrate that flow sorting on the basis of CD59 expression can effectively purify RPE and deplete stem cells, resulting in a population free from stem cell impurity. This purification helps to ensure removal of stem cells and hence increases the safety of cells that may be used for clinical transplantation. This strategy can potentially be applied to other pluripotent stem cell-derived material and help mitigate concerns of using such cells for therapy.
authors: Pfizer Neuroscience and Pain Research Unit, The Portway, Granta Park, Great Abington, Cambridge, CB21 6GS, UK. parulc83@gmail.com.
authors: Parul
authors: P
authors: Choudhary
authors: Present Address: AR-UK Drug Discovery Institute, University College London, London, WC1E 6BT, UK.
authors: Paul John
authors: PJ
authors: Whiting
mesh_terms: D015415:Biomarkers / Q000378:metabolism; D018957:CD59 Antigens / Q000378:metabolism; D002460:Cell Line; D064987:Cell- and Tissue-Based Therapy / Q000379:methods; D053595:Embryonic Stem Cells / Q000166:cytology* / Q000378:metabolism; D006801:Humans; D008268:Macular Degeneration / Q000378:metabolism / Q000628:therapy; D055213:Retinal Pigment Epithelium / Q000166:cytology* / Q000378:metabolism; D012449:Safety
chemical_list: D015415:Biomarkers; D018957:CD59 Antigens
keywords: Age-related macular degeneration; Cell surface markers; Embryonic stem cells; Flow sorting; Retinal pigment epithelium