Development of virus vectors for gene therapy of beta chain hemoglobinopathies: flanking with a chromatin insulator reduces gamma-globin gene silencing in vivo.

@article{Emery2002DevelopmentOV,
  title={Development of virus vectors for gene therapy of beta chain hemoglobinopathies: flanking with a chromatin insulator reduces gamma-globin gene silencing in vivo.},
  author={David W. Emery and Evangelia Yannaki and Julie Tubb and Tamon Nishino and Qiliang Li and G. Stamatoyannopoulos},
  journal={Blood},
  year={2002},
  volume={100 6},
  pages={2012-9}
}
We have previously described the development of oncoretrovirus vectors for human gamma-globin using a truncated beta-globin promoter, modified gamma-globin cassette, and alpha-globin enhancer. However, one of these vectors is genetically unstable, and both vectors exhibit variable expression patterns in cultured cells, common characteristics of oncoretrovirus vectors for globin genes. To address these problems, we identified and removed the vector sequences responsible for genetic instability… CONTINUE READING

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