Article
Successful therapeutic effect in a mouse model of erythropoietic protoporphyria by partial genetic correction and fluorescence-based selection of hematopoietic cells.
Gene therapy - 1 Apr 2001
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
Abstract excerpt
Erythropoietic protoporphyria is characterized clinically by skin photosensitivity and biochemically by a ferrochelatase deficiency resulting in an excessive accumulation of photoreactive protoporphyrin in erythrocytes, plasma and other organs. The availability of the Fech(m1Pas)/Fech(m1Pas) murine model allowed us to test a gene therapy protocol to correct the porphyric phenotype. Gene therapy was performed by...
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