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Another study showed that knock-down of BCL11A mRNA expression using an RNA interference strategy that was delivered into autologous CD34 + HSPC via a third-generation SIN lentiviral vector was safe and feasible to treat sickle cell disease, which is also caused by mutation of the -globin gene (Fig

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Clearly, in studies designed to establish adequate/normal serum MAA adult values, it is advisable to evaluate renal function using serum creatinine, especially in older adults