Gene Therapy of a Mouse Model of Protoporphyria with a Self-Inactivating Erythroid-Specific Lentiviral Vector without Preselection
Open Access
- 31 October 2001
- journal article
- Published by Elsevier BV in Molecular Therapy
- Vol. 4 (4), 331-338
- https://doi.org/10.1006/mthe.2001.0467
Abstract
No abstract availableKeywords
This publication has 30 references indexed in Scilit:
- Correction of Deficient CD34+ Cells from Peripheral Blood after Mobilization in a Patient with Congenital Erythropoietic PorphyriaMolecular Therapy, 2001
- Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 DiseaseScience, 2000
- Fluorescence-based selection of retrovirally transduced cells in congenital erythropoietic porphyria: direct selection based on the expression of the therapeutic geneThe Journal of Gene Medicine, 1999
- Long-term cure of the photosensitivity of murine erythropoietic protoporphyria by preselective gene therapyNature Medicine, 1999
- Congenital Erythropoietic Porphyria: Prolonged High-Level Expression and Correction of the Heme Biosynthetic Defect by Retroviral-Mediated Gene Transfer into Porphyric and Erythroid CellsMolecular Genetics and Metabolism, 1998
- Molecular defects in ferrochelatase in patients with protoporphyria requiring liver transplantation.JCI Insight, 1998
- Correction of the Enzyme Defect in Cultured Congenital Erythropoietic Porphyria Disease Cells by Retrovirus-Mediated Gene TransferHuman Gene Therapy, 1995
- Ferrochelatase Structural Mutant (Fechm1Pas) in the House MouseGenomics, 1993
- Erythropoietic protoporphyria in the house mouse. A recessive inherited ferrochelatase deficiency with anemia, photosensitivity, and liver disease.JCI Insight, 1991
- Uroporphyrinogen Decarboxylase Structural Mutant (Gly281→Glu) in a Case of PorphyriaScience, 1986