Modeling of congenital erythropoietic po
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Elodie Robert-Richard; Magalie Lalanne; Isabelle Lamrissi-Garcia; VΓ©ronique Guyo
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Article
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2010
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John Wiley and Sons
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English
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## Abstract ## Background Congenital erythropoietic porphyria (CEP) is a severe autosomal recessive disorder characterized by a deficiency in uroporphyrinogen III synthase (UROS), the fourth enzyme of the heme biosynthetic pathway. We recently demonstrated the definitive cure of a murine model of