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Research
Nature Biotechnology 24, 89–94 (1 January 2006) | doi:10.1038/nbt1176
A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference
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Abstract
The application of RNA interference (RNAi) to stem cell–based therapies will require highly specific and lineage-restricted gene silencing. Here we show the feasibility and therapeutic potential of coregulating transgene expression and RNAi in hematopoietic stem cells.
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