Effective Gene Therapy of Mice with Congenital Erythropoietic Porphyria Is Facilitated by a Survival Advantage of Corrected Erythroid Cells

Author:

Robert-Richard Elodie,Moreau-Gaudry François,Lalanne Magalie,Lamrissi-Garcia Isabelle,Cario-André Muriel,Guyonnet-Dupérat Véronique,Taine Laurence,Ged Cécile,de Verneuil Hubert

Publisher

Elsevier BV

Subject

Genetics(clinical),Genetics

Reference54 articles.

1. Disorders of heme biosynthesis: X-linked sideroblastic anemia and the porphyrias;Anderson,2001

2. Congenital erythropoietic porphyria;de Verneuil,2003

3. Uroporphyrinogen III cosynthase in human congenital erythropoietic porphyria;Romeo;Biochemistry,1969

4. Point mutations in the uroporphyrinogen III synthase gene in congenital erythropoietic porphyria;Deybach;Blood,1990

5. Congenital erythropoietic porphyria: Identification and expression of 10 mutations in the uroporphyrinogen III synthase gene;Xu;J. Clin. Invest.,1995

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