Figure 7.
Figure 7. Schematic representation of HIV-1–based, lentiviral globin expression cassettes utilized to correct murine models of β-thalassemia and sickle cell disease. The mLARβΔγV5 globin expression cassette is compared with those contained in TNS9, βA-T87Q, and βAS.31,2,5 DNA fragments containing the HS sites and flanking sequences appear as open horizontal rectangles, the solid horizontal arrow indicates the β-globin promoter, and the hatched horizontal arrows indicate the genomic β- or γ-globin sequences contained in the mature mRNA. The solid lines connecting the 3 arrows represent intronic sequences; all of the vectors utilized a deletion in intron 2 to improve vector performance. The solid gray rectangle indicates the 3′β-globin enhancer sequence. The fragment sizes of the DNA elements derived from the β-globin LCR that contain the indicated HS sites are shown below each horizontal rectangle, whereas the GenBank coordinates (accession number U01317) of the sequences are shown above each rectangle.

Schematic representation of HIV-1–based, lentiviral globin expression cassettes utilized to correct murine models of β-thalassemia and sickle cell disease. The mLARβΔγV5 globin expression cassette is compared with those contained in TNS9, βA-T87Q, and βAS.31,2,5 DNA fragments containing the HS sites and flanking sequences appear as open horizontal rectangles, the solid horizontal arrow indicates the β-globin promoter, and the hatched horizontal arrows indicate the genomic β- or γ-globin sequences contained in the mature mRNA. The solid lines connecting the 3 arrows represent intronic sequences; all of the vectors utilized a deletion in intron 2 to improve vector performance. The solid gray rectangle indicates the 3′β-globin enhancer sequence. The fragment sizes of the DNA elements derived from the β-globin LCR that contain the indicated HS sites are shown below each horizontal rectangle, whereas the GenBank coordinates (accession number U01317) of the sequences are shown above each rectangle.

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