US 11,701,435 B2
Therapeutic retroviral vectors for gene therapy
Philippe Louis Leboulch, Bangkok (TH); Robert Pawliuk, Medford, MA (US); and Karen Westerman, Reading, MA (US)
Assigned to bluebird bio, Inc., Somerville, MA (US); and Massachusetts Institute of Technology, Cambridge, MA (US)
Filed by bluebird bio, Inc., Cambridge, MA (US); and Massachusetts Institute of Technology, Cambridge, MA (US)
Filed on Mar. 16, 2020, as Appl. No. 16/820,066.
Application 16/130,845 is a division of application No. 14/700,021, filed on Apr. 29, 2015, granted, now 10,105,451, issued on Oct. 23, 2018.
Application 14/700,021 is a division of application No. 13/242,815, filed on Sep. 23, 2011, granted, now 9,068,199, issued on Jun. 30, 2015.
Application 16/820,066 is a continuation of application No. 16/130,845, filed on Sep. 13, 2018, granted, now 10,632,212.
Application 13/242,815 is a continuation of application No. 12/976,078, filed on Dec. 22, 2010, abandoned.
Application 12/976,078 is a continuation of application No. 11/150,785, filed on Jun. 9, 2005, granted, now 7,901,671, issued on Mar. 8, 2011.
Application 11/150,785 is a continuation of application No. PCT/US03/39526, filed on Dec. 11, 2003.
Claims priority of provisional application 60/513,312, filed on Oct. 21, 2003.
Claims priority of provisional application 60/475,822, filed on Jun. 4, 2003.
Claims priority of provisional application 60/433,321, filed on Dec. 13, 2002.
Prior Publication US 2020/0368370 A1, Nov. 26, 2020
This patent is subject to a terminal disclaimer.
Int. Cl. A01N 63/00 (2020.01); C12N 15/79 (2006.01); C07H 21/04 (2006.01); A61K 48/00 (2006.01); C07K 14/805 (2006.01); A01K 67/027 (2006.01); C12N 15/85 (2006.01); C12N 15/86 (2006.01); A61K 38/42 (2006.01); C12N 15/867 (2006.01); C12N 15/63 (2006.01)
CPC A61K 48/0058 (2013.01) [A01K 67/0278 (2013.01); A61K 38/42 (2013.01); C07K 14/805 (2013.01); C12N 15/8509 (2013.01); C12N 15/86 (2013.01); A01K 2207/15 (2013.01); A01K 2217/00 (2013.01); A01K 2227/105 (2013.01); A01K 2267/03 (2013.01); A61K 48/00 (2013.01); C07H 21/04 (2013.01); C12N 15/63 (2013.01); C12N 15/867 (2013.01); C12N 2510/00 (2013.01); C12N 2740/15043 (2013.01); C12N 2740/16011 (2013.01); C12N 2740/16043 (2013.01); C12N 2830/008 (2013.01); C12N 2830/40 (2013.01)] 20 Claims
 
1. A hematopoietic stem cell comprising a self-inactivating (SIN) lentiviral vector comprising:
a) a human immunodeficiency virus 1 (HIV-1) 5′ long terminal repeat (LTR), wherein the promoter of the HIV-1 5′ LTR is replaced with a heterologous promoter;
b) an RNA export element;
c) a central polypurine tract or DNA FLAP (cPPT/FLAP);
d) a human β-globin promoter;
e) a human β-globin locus control region (LCR), wherein the β-globin promoter and β-globin LCR are operatively linked to a polynucleotide encoding a human globin;
f) a human β-globin 3′ enhancer element; and
g) a HIV-1 3′ LTR, wherein the 3′ LTR comprises at least one deletion compared to the wild-type HIV-1 3′ LTR.