High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors.

@article{MoreauGaudry2001HighlevelEG,
  title={High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors.},
  author={François Moreau-Gaudry and Ping Xia and Guosheng Jiang and Natalya Perelman and Gerhard Bauer and James Ellis and Kathy H Surinya and Fulvio Mavilio and Chia-Ning Shen and Punam Malik},
  journal={Blood},
  year={2001},
  volume={98 9},
  pages={2664-72}
}
Use of oncoretroviral vectors in gene therapy for hemoglobinopathies has been impeded by low titer vectors, genetic instability, and poor expression. Fifteen self- inactivating (SIN) lentiviral vectors using 4 erythroid promoters in combination with 4 erythroid enhancers with or without the woodchuck hepatitis virus postregulatory element (WPRE) were generated using the enhanced green fluorescent protein as a reporter gene. Vectors with high erythroid-specific expression in cell lines were… CONTINUE READING

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Successful gene therapy in a murine model of erythropoietic protoporphyria with a self inactivating erythroid-specific lentiviral vector [abstract

F Moreau-Gaudry, E Richard, P Xia
Blood. 2000;96:590a • 2001

cessful gene therapy in a murine model of eryth - ropoietic protoporphyria with a self inactivating erythroid - specific lentiviral vector [ abstract ]

F Moreau-Gaudry, E Richard, P Xia
Blood • 2000

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