Metabolic Correction of Congenital Erythropoietic Porphyria with iPSCs Free of Reprogramming Factors

Congenital erythropoietic porphyria (CEP) is due to a deficiency in the enzymatic activity of uroporphyrinogen III synthase (UROS); such a deficiency leads to porphyrin accumulation and results in skin lesions and hemolytic anemia. CEP is a candidate for retrolentivirus-mediated gene therapy, but re...

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Auteurs principaux: Bedel, Aurélie, Taillepierre, Miguel, Guyonnet-Duperat, Véronique, Lippert, Eric, Dubus, Pierre, Dabernat, Sandrine, Mautuit, Thibaud, Cardinaud, Bruno, Pain, Catherine, Rousseau, Benoît, Lalanne, Magalie, Ged, Cécile, Duchartre, Yann, Richard, Emmanuel, de Verneuil, Hubert, Moreau-Gaudry, François
Format: Artigo
Langue:Inglês
Publié: Elsevier 2012
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Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC3397263/
https://ncbi.nlm.nih.gov/pubmed/22795135
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ajhg.2012.05.026
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