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Induced-pluripotent stem (iPS)-derived RPE cells from patients augment gene therapy for recessive RP

Researchers based at Columbia University, New York, USA, have published novel research illustrating both a putative gene therapy treatment for recessive retinitis pigmentosa (RP), and a potentially beneficial model for translating lab based findings to clinical trials more efficiently. The research, published in the Nature journal, Molecular Therapy (2014; 22 9, 1688—1697), indicates that patient specific induced-pluripotent stem cells can provide a valuable bridge between lab-based testing and human clinical trials. The ophthalmic and stem cell research group used patient induced-pluripotent stem (iPS) derived RPE cells to test a gene therapy approach in the very type of human cells that would ultimately receive the clinical therapy. The authors of the study hope that “patient-specific iPS cells will help to decrease patient risk, clarify disease mechanisms, bypass problems related to differences among species that arise when using animal models, and reduce the cost of clinical trials”.