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

  • US 7,541,179 B2
  • Filed: 07/01/2002
  • Issued: 06/02/2009
  • Est. Priority Date: 06/29/2001
  • Status: Active Grant
First Claim
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1. A recombinant vector comprising a nucleic acid encoding a functional globin operably linked to a 3.2-kb nucleotide fragment which consists essentially of three contiguous nucleotide fragments obtainable from a human β

  • -globin locus control region (LCR), the three fragments being a BstXI and SnaBI HS2-spanning nucleotide fragment of said LCR, a BamHI and HindIII HS3-spanning nucleotide fragment of said LCR and a BamHI and BanII HS4-spanning nucleotide fragment of said LCR, said vector providing expression of the globin in a mammal in vivo.

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