Figure 1.
Figure 1. LentiGlobin vector and experimental design. (A) Diagram of the human β-globin (βA-T87Q) lentiviral vector (LentiGlobin; LG). The 3′ β-globin enhancer, the 372-bp IVS2 deletion, the βA-T87Q mutation (ACA[Thr] to CAG[Gln]) and DNase I Hypersensitive Sites (HS) 2, HS3, and HS4 of the human β-globin Locus Control Region (LCR) are indicated. Safety modifications including the 2 stop codons in the ψ+ signal, the 400-bp deletion in the U3 of the right HIVLTR, the rabbit β-globin plyA signal, and the 2 × 250-bp cHS4 chromatin insulators are indicated. HIVLTR indicates human immunodeficiency type-1 virus long terminal repeat; ψ+, packaging signal; cPPT Map, central polypurine tract; RRE, Rev-responsive element; βp, human β-globin promoter; and ppt, polypurine tract.

LentiGlobin vector and experimental design. (A) Diagram of the human β-globin (βA-T87Q) lentiviral vector (LentiGlobin; LG). The 3′ β-globin enhancer, the 372-bp IVS2 deletion, the βA-T87Q mutation (ACA[Thr] to CAG[Gln]) and DNase I Hypersensitive Sites (HS) 2, HS3, and HS4 of the human β-globin Locus Control Region (LCR) are indicated. Safety modifications including the 2 stop codons in the ψ+ signal, the 400-bp deletion in the U3 of the right HIVLTR, the rabbit β-globin plyA signal, and the 2 × 250-bp cHS4 chromatin insulators are indicated. HIVLTR indicates human immunodeficiency type-1 virus long terminal repeat; ψ+, packaging signal; cPPT Map, central polypurine tract; RRE, Rev-responsive element; βp, human β-globin promoter; and ppt, polypurine tract.

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