Gene therapy of a mouse model of protoporphyria with a self-inactivating erythroid-specific lentiviral vector without preselection.

@article{Richard2001GeneTO,
  title={Gene therapy of a mouse model of protoporphyria with a self-inactivating erythroid-specific lentiviral vector without preselection.},
  author={Emmanuel Richard and Michael Anthony Mendez and Fr{\'e}deric Mazurier and C{\'e}line Morel and Pierre Costet and Ping Xia and Antonio Fontanellas and Fabien G{\'e}ronimi and Muriel Cario-Andre and Laurence Taine and C{\'e}cile Ged and Punam Malik and Hubert de Verneuil and François Moreau-Gaudry},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2001},
  volume={4 4},
  pages={331-8}
}
Successful treatment of blood disorders by gene therapy has several complications, one of which is the frequent lack of selective advantage of genetically corrected cells. Erythropoietic protoporphyria (EPP), caused by a ferrochelatase deficiency, is a good model of hematological genetic disorders with a lack of spontaneous in vivo selection. This disease is characterized by accumulation of protoporphyrin in red blood cells, bone marrow, and other organs, resulting in severe skin… CONTINUE READING

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