Optimization of recombinant adeno-associated viral vectors for human beta-globin gene transfer and transgene expression.


Therapeutic levels of expression of the beta-globin gene have been difficult to achieve with conventional retroviral vectors without the inclusion of DNase I-hypersensitive site (HS2, HS3, and HS4) enhancer elements. We generated recombinant adeno-associated viral (AAV) vectors carrying an antisickling human beta-globin gene under the control of either the… (More)
DOI: 10.1089/hum.2007.173


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