US 7,541,179 B2
	Vector encoding human globin gene and use thereof in treatment of hemoglobinopathies
Michel Sadelain, New York, N.Y. (US); Stefano Rivella, New York, N.Y. (US); Chad May, New York, N.Y. (US); and Joseph Bertino, New York, N.Y. (US)
Assigned to Memorial Sloan-Kettering Cancer Center, New York, N.Y. (US)
Filed on Jul. 01, 2002, as Appl. No. 10/188,221.
Claims priority of provisional application 60/301861, filed on Jun. 29, 2001.
Claims priority of provisional application 60/302852, filed on Jul. 02, 2001.
Prior Publication US 2003/0022303 A1, Jan. 30, 2003
Int. Cl. C12N 15/00 (2006.01); C12N 15/10 (2006.01); C12N 15/67 (2006.01); C12N 7/01 (2006.01)

U.S. Cl. 435—320.1  [435/69.1; 536/24.1; 536/24.2; 424/93.2]

24 Claims

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.