| Successful
Sickle Gene Therapy |
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| l | Pawliuk
et al: Science 2001 |
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| l | b globin gene correction in two |
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| mouse
models of sickle cell |
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| l | Lentiviral
vectors with extensive b |
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| -LCR
elements used |
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| l | Gene-modified
cells produce b globin |
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| in
vivo |
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| l | Correction
of sickle phenotype |
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