Article
Stable in vivo expression of glucose-6-phosphate dehydrogenase (G6PD) and rescue of G6PD deficiency in stem cells by gene transfer.
Blood - 15 Dec 2000
Rovira A, De Angioletti M, Camacho-Vanegas O, Liu D, Rosti V, Gallardo H F, Notaro R, Sadelain M, Luzzatto L
Abstract excerpt
Many mutations of the housekeeping gene encoding glucose-6-phosphate dehydrogenase (G6PD) cause G6PD deficiency in humans. Some underlie severe forms of chronic nonspherocytic hemolytic anemia (CNSHA) for which there is no definitive treatment. By using retroviral vectors pseudotyped with the vesicular stomatitis virus G glycoprotein that harbor the human G6PD (hG6PD) complementary DNA, stable and lifelong...
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