Modeling of congenital erythropoietic porphyria by RNA interference: a new tool for preclinical gene therapy evaluation.

Robert-Richard, Elodie; Lalanne, Magalie; Lamrissi-Garcia, Isabelle; Guyonnet-Duperat, Véronique; Richard, Emmanuel; Pitard, Vincent; Mazurier, Fréderic; Moreau-Gaudry, François; Ged, Cécile; de Verneuil, Hubert

Robert-Richard, Elodie; Lalanne, Magalie; Lamrissi-Garcia, Isabelle; Guyonnet-Duperat, Véronique; Richard, Emmanuel; Pitard, Vincent; Mazurier, Fréderic; Moreau-Gaudry, François; Ged, Cécile; de Verneuil, Hubert.

Affiliation

Robert-Richard E; INSERM U876, Bordeaux, France.

J Gene Med ; 12(8): 637-46, 2010 Aug.

Article in En | MEDLINE | ID: mdl-20586119

Subject(s)

Genetic Therapy/methods; Porphyria, Erythropoietic/therapy; RNA Interference; Animals; Disease Models, Animal; Hematopoietic Stem Cells/metabolism; Humans; K562 Cells; Lentivirus/genetics; Lentivirus/metabolism; Mice; Porphyria, Erythropoietic/enzymology; Porphyria, Erythropoietic/genetics; Uroporphyrinogen III Synthetase/genetics; Uroporphyrinogen III Synthetase/metabolism

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Porphyria, Erythropoietic / RNA Interference Type of study: Evaluation_studies Limits: Animals / Humans Language: En Journal: J Gene Med Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2010 Document type: Article Affiliation country: France Country of publication: United kingdom

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