Figure 6.
Figure 6. The lentivirus vector use for the correction of sickle cell in transgenic mice. / Diagram of the βT27Q-globin provirus. / Abbreviations: HIV LTR, human immune deficiency type-1 virus long terminal repeat; +, packaging signal; cPPT/flap or central polypurine tract/DNA flap; RRE or Rev-responsive element; P, β-globin promoter; ppt, polypurine tract. / The 3′ β-globin enhancer, the 372 bp IVS2 deletion, the βT87Q mutation and sections of DnaseI hypersensitive sites (HS) 2, HS3, and HS4 of the β-globin LCR are indicated. / The cPPT/flap and the RRE element help in the export of the mRNA from the nucleus to the cytoplasma. / This vector integrates with an average of 3 copies per genome that is higher than previous vectors. / This vector has given no indication of silencing over time. / Reprinted with permission from Pawliuk R, Westerman KA, Fabry ME, et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science. 2000;294:2368–2371. / Abbreviations: LCR, locus control region, 5′ to the β-like gene cluster, involved in the development-appropriate globin gene expression; IVS2, intervening sequence 2.

The lentivirus vector use for the correction of sickle cell in transgenic mice.

Diagram of the βT27Q-globin provirus.

Abbreviations: HIV LTR, human immune deficiency type-1 virus long terminal repeat; +, packaging signal; cPPT/flap or central polypurine tract/DNA flap; RRE or Rev-responsive element; P, β-globin promoter; ppt, polypurine tract.

The 3′ β-globin enhancer, the 372 bp IVS2 deletion, the βT87Q mutation and sections of DnaseI hypersensitive sites (HS) 2, HS3, and HS4 of the β-globin LCR are indicated.

The cPPT/flap and the RRE element help in the export of the mRNA from the nucleus to the cytoplasma.

This vector integrates with an average of 3 copies per genome that is higher than previous vectors.

This vector has given no indication of silencing over time.

Reprinted with permission from

Pawliuk R, Westerman KA, Fabry ME, et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science. 2000;294:2368–2371.

Abbreviations: LCR, locus control region, 5′ to the β-like gene cluster, involved in the development-appropriate globin gene expression; IVS2, intervening sequence 2.

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