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Metabolic correction of congenital erythropoietic porphyria with iPSCs free of reprogramming factors.
Bedel A, Taillepierre M, Guyonnet-Duperat V, Lippert E, Dubus P, Dabernat S, Mautuit T, Cardinaud B, Pain C, Rousseau B, Lalanne M, Ged C, Duchartre Y, Richard E, de Verneuil H, Moreau-Gaudry F. Bedel A, et al. Among authors: richard e. Am J Hum Genet. 2012 Jul 13;91(1):109-21. doi: 10.1016/j.ajhg.2012.05.026. Am J Hum Genet. 2012. PMID: 22795135 Free PMC article.
Successful therapeutic effect in a mouse model of erythropoietic protoporphyria by partial genetic correction and fluorescence-based selection of hematopoietic cells.
Fontanellas A, Mendez M, Mazurier F, Cario-André M, Navarro S, Ged C, Taine L, Géronimi F, Richard E, Moreau-Gaudry F, Enriquez De Salamanca R, de Verneuil H. Fontanellas A, et al. Among authors: richard e. Gene Ther. 2001 Apr;8(8):618-26. doi: 10.1038/sj.gt.3301427. Gene Ther. 2001. PMID: 11320408
637 results