Vector encoding human globin gene and use thereof in treatment of hemoglobinopathies

  • US 20030022303A1
  • Filed: 07/01/2002
  • Published: 01/30/2003
  • Est. Priority Date: 06/29/2001
  • Status: Active Grant
First Claim
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1. A recombinant lentiviral vector comprising:

  • (a) a region comprising a functional globin gene; and

    (b) large portions of the β

    -globin locus control region, which include DNase I hypersensitive sites HS2, HS3 and HS4, said vector providing expression of the globin gene when introduced into a mammal in vivo.

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