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.
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