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

Molecular Therapy

Volumn 4, Issue 4, 2001, Pages 331-338

  Richard, Emmanuel ^a   Mendez, Manuel ^a   Mazurier, Fréderic ^a   Morel, Carine ^a   Costet, Pierre ^a   Xia, Ping ^b   Fontanellas, Antonio ^a   Geronimi, Fabien ^a   Cario André, Muriel ^a   Taine, Laurence ^a   Ged, Cecile ^a   Malik, Punam ^b   De Verneuil, Hubert ^a   Moreau Gaudry, François ^a  

^a UNIVERSITÉ DE BORDEAUX   (France)

^b CHILDREN S HOSPITAL LOS ANGELES   (United States)

Author keywords

Ankyrin 1 promoter; Erythroid specificity; Ferrochelatase; Gene therapy; Gene transfer; Hematopoietic stem cell; Lentiviral vectors; Porphyria; Skin photosensitivity

Indexed keywords

ALPHA GLOBIN; ANKYRIN; ANKYRIN 1; COMPLEMENTARY DNA; FERROCHELATASE; PROTOPORPHYRIN; UNCLASSIFIED DRUG; VIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BLOOD DISEASE; BONE MARROW CELL; CELL LINEAGE; CELL SELECTION; CLINICAL PROTOCOL; CONTROLLED STUDY; DISEASE SEVERITY; DONOR; DRUG INACTIVATION; DRUG SYNTHESIS; ENHANCER REGION; ENZYME DEFICIENCY; ERYTHROCYTE; ERYTHROID CELL; ERYTHROPOIETIC PROTOPORPHYRIA; FEMALE; GENE THERAPY; GENETIC DISORDER; GENETIC PROCEDURES; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; IN VIVO STUDY; LENTIVIRINAE; LETHALITY; MALE; MOUSE; NONHUMAN; PHOTOSENSITIVITY; PROMOTER REGION; RECIPIENT;

ANIMALIA; LENTIVIRUS;

EID: 0034756572     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2001.0467     Document Type: Article

References (37)

1. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
2. Hematologic aspects of the porphyrias
3. Uroporphyrinogen decarboxylase structural mutant (Gly281→Glu) in a case of porphyria
4. Metabolic correction of congenital erythropoietic porphyria by retrovirus-mediated gene transfer into Epstein-Barr virus-transformed B-cell lines
5. Correction of the enzyme defect in cultured congenital erythropoietic porphyria disease cells by retrovirus-mediated gene transfer
6. Congenital erythropoietic porphyria: Prolonged high-level expression and correction of the heme biosynthetic defect by retroviral-mediated transfer into porphyric and erythroid cells
7. Fluorescence-based selection of retrovirally transduced cells in congenital erythropoietic porphyria: Direct selection based on the expression of the therapeutic gene
8. + cells from peripheral blood after mobilization in a patient with congenital erythropoietic porphyria
9. Erythropoietic protoporphyria in the house mouse. A recessive inherited ferrochelatase deficiency with anemia, photosensitivity, and liver disease
10. Ferrochelatase structural mutant (Fechm 1 Pas) in the house mouse
11. Inheritance in erythropoietic protoporphyria: A common wild-type ferrochelatase allelic variant with low expression accounts for clinical manifestation
12. Molecular defects in ferrochelatase in patients with protoporphyria requiring liver transplantation
13. Long-term cure of the photosensitivity of murine erythropoietic protoporphyria by preselective gene therapy
14. Successful therapeutic effect in a mouse model of erythropoietic protoporphyria by partial genetic correction and fluorescence-based selection of hematopoietic cells
15. Immune response to green fluorescent protein: Implications for gene therapy
16. Induction of cytotoxic T lymphocyte and antibody responses to enhanced green fluorescent protein following transplantation of transduced CD34(+) hematopoietic cells
17. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
18. HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells
19. Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors
20. Lentiviral gene transfer into primary and secondary NOD/SCID repopulating cells
21. Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors
22. Enhanced transgene expression in cord blood CD34(+)-derived hematopoietic cells, including developing T cells and nod/scid mouse repopulating cells, following transduction with modified trip lentiviral vectors
23. Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin
24. Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors
25. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery
26. High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors
27. Derepression of human embryonic ζ-globin promoter by a locus-control region sequence
28. Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors
29. Reversion of hepatobiliary alterations by bone marrow transplantation in a murine model of erythropoietic protoporphyria
30. Structure and transcriptional regulation of the mouse ferrochelatase gene
31. Analysis of ferrochelatase expression during hematopoietic development of embryonic stem cells
32. Bone-marrow transplantation for congenital erythropoietic porphyria
33. Correction of congenital erythropoietic porphyria by bone marrow transplantation
34. Congenital erythropoietic porphyria successfully treated by allogeneic bone marrow transplantation
35. Long-term expression of γ-globin mRNA in mouse erythrocytes from retrovirus vectors containing the human γ-globin gene fused to the ankyrin-1 promoter
36. Long term transduction and lineage-specific expression of self-inactivating (SIN) lentiviral vectors in murine hematopoietic stem cells
37. A simple ferrochelatase assay