作者: Tina Guanting Qiu
DOI: 10.1038/S41536-019-0081-8
关键词: Macular degeneration 、 Embryonic Germ Cells 、 Retina 、 Clinical trial 、 Regenerative medicine 、 Bioinformatics 、 Embryonic stem cell 、 Medicine 、 Retinal pigment epithelium 、 Transplantation
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.