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Modeling of congenital erythropoietic porphyria by RNA interference: a new tool for preclinical gene therapy evaluation |
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| Authors: | Elodie Robert‐Richard Magalie Lalanne Isabelle Lamrissi‐Garcia Véronique Guyonnet‐Duperat Emmanuel Richard Vincent Pitard Fréderic Mazurier François Moreau‐Gaudry Cécile Ged Hubert de Verneuil |
| Affiliation: | 1. INSERM U876, Bordeaux, France;2. Université Victor Segalen Bordeaux 2, Bordeaux, France;3. CNRS UMR 5164, IFR 66, Bordeaux, France |
| Abstract: | |
| Keywords: | congenital erythropoietic porphyria gene therapy lentiviral vectors RNAi shRNA |