Transplantation of human embryonic stem cell-derived retinal pigment epithelial cells (MA09-hRPE) in macular degeneration.

作者: Tina Guanting Qiu

DOI: 10.1038/S41536-019-0081-8

关键词: Macular degenerationEmbryonic Germ CellsRetinaClinical trialRegenerative medicineBioinformaticsEmbryonic stem cellMedicineRetinal pigment epitheliumTransplantation

摘要: The use of human embryonic stem cell (hESC)-derived Retinal Pigment Epithelium (RPE) transplants has advanced dramatically in different forms for clinical application macular degeneration. This review focuses on the first generation hESC-RPE line, named as “MA09-hRPE” by Astellas Institute Regenerative Medicine (AIRM), and its therapeutic human, which evaluated safety efficacy MA09-hRPE line transplanted patients with project marks milestone overcoming ethical hurdles oncogenic concerns associated an cell-derived line. Through in-depth, evidence-based analysis along other lines, this aims to draw attention key technical challenges pertinent a biologically competent distill four prognostic factors residing host retina, concurrently determine outcomes visual benefits. Given that technology is still at infancy use, new regulatory path could aid validation through small cohort, adaptive trials accelerate product development toward commercialization. These strategic insights will be invaluable help both academia industry, collaboratively shorten steep learning curve, reduce large expenditures spent unnecessary lengthy trials.

参考文章(35)
Guanting Qiu, Magdalene J. Seiler, Cathy Mui, Shinichi Arai, Robert B. Aramant, Eugene de Juan, SriniVas Sadda, Photoreceptor differentiation and integration of retinal progenitor cells transplanted into transgenic rats. Experimental Eye Research. ,vol. 80, pp. 515- 525 ,(2005) , 10.1016/J.EXER.2004.11.001
Kentaro Azuma, Regulatory Landscape of Regenerative Medicine in Japan Stem cell reports. ,vol. 1, pp. 118- 128 ,(2015) , 10.1007/S40778-015-0012-6
Guanting Qiu, Magdalene J. Seiler, Biju B. Thomas, Kebin Wu, Michael Radosevich, SriniVas R. Sadda, Revisiting nestin expression in retinal progenitor cells in vitro and after transplantation in vivo Experimental Eye Research. ,vol. 84, pp. 1047- 1059 ,(2007) , 10.1016/J.EXER.2007.01.014
Ilene K. Sugino, Qian Sun, Jianqiu Wang, Celia F. Nunes, Noounanong Cheewatrakoolpong, Aprille Rapista, Adam C. Johnson, Christopher Malcuit, Irina Klimanskaya, Robert Lanza, Marco A. Zarbin, Comparison of FRPE and Human Embryonic Stem Cell–Derived RPE Behavior on Aged Human Bruch's Membrane Investigative Ophthalmology & Visual Science. ,vol. 52, pp. 4979- 4997 ,(2011) , 10.1167/IOVS.10-5386
Won Kyung Song, Kyung-Mi Park, Hyun-Ju Kim, Jae Ho Lee, Jinjung Choi, So Young Chong, Sung Han Shim, Lucian V. Del Priore, Robert Lanza, Treatment of Macular Degeneration Using Embryonic Stem Cell-Derived Retinal Pigment Epithelium: Preliminary Results in Asian Patients Stem cell reports. ,vol. 4, pp. 860- 872 ,(2015) , 10.1016/J.STEMCR.2015.04.005
Bartosz Różanowski, Janice Burke, Tadeusz Sarna, Małgorzata Różanowska, The pro-oxidant effects of interactions of ascorbate with photoexcited melanin fade away with aging of the retina Photochemistry and Photobiology. ,vol. 84, pp. 658- 670 ,(2008) , 10.1111/J.1751-1097.2007.00291.X
Steven D Schwartz, Jean-Pierre Hubschman, Gad Heilwell, Valentina Franco-Cardenas, Carolyn K Pan, Rosaleen M Ostrick, Edmund Mickunas, Roger Gay, Irina Klimanskaya, Robert Lanza, Embryonic stem cell trials for macular degeneration: a preliminary report The Lancet. ,vol. 379, pp. 713- 720 ,(2012) , 10.1016/S0140-6736(12)60028-2
Bruno Diniz, Padmaja Thomas, Biju Thomas, Ramiro Ribeiro, Yuntao Hu, Rodrigo Brant, Ashish Ahuja, Danhong Zhu, Laura Liu, Michael Koss, Mauricio Maia, Gerald Chader, David R. Hinton, Mark S. Humayun, Subretinal implantation of retinal pigment epithelial cells derived from human embryonic stem cells: improved survival when implanted as a monolayer. Investigative Ophthalmology & Visual Science. ,vol. 54, pp. 5087- 5096 ,(2013) , 10.1167/IOVS.12-11239
Christopher Thomas Scott, David Magnus, Wrongful Termination: Lessons From the Geron Clinical Trial Stem Cells Translational Medicine. ,vol. 3, pp. 1398- 1401 ,(2014) , 10.5966/SCTM.2014-0147
Kazuto Kato, Jonathan Kimmelman, Jason Robert, Douglas Sipp, Jeremy Sugarman, None, Ethical and policy issues in the clinical translation of stem cells: report of a focus session at the ISSCR Tenth Annual Meeting. Cell Stem Cell. ,vol. 11, pp. 765- 767 ,(2012) , 10.1016/J.STEM.2012.11.004