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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

Selda Samakoglu , Leszek Lisowski , Tulin Budak-Alpdogan , Yelena Usachenko , Santina Acuto , Rosalba Di Marzo , Aurelio Maggio , Ping Zhu , John F. Tisdale , Isabelle Rivi|[egrave]|re & Michel Sadelain

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.