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Urbinati et al., 2006 - Google Patents

467. Lentiviral Vector (LV) Non-Coding Elements That Impart High Titers and Expression to β-Globin (hβ) Cassettes

Urbinati et al., 2006

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Document ID
2703278792762554924
Author
Urbinati F
Xia P
Perumbeti A
Xia M
Corsinotti A
Zarzuela A
Perelman N
Malik P
Publication year
Publication venue
Molecular Therapy

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Snippet

Thalassemias are the most common monogenic defects that result from absent/reduced hβ in RBC. However, the complexities of the hβ gene and its regulatory elements have been significant obstacles for optimization of gene therapy approaches using γ-oncoretroviral …
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6072—Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses
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