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Successful correction of the human beta-thalassemia major phenotype using a lentiviral vector.
Puthenveetil G, Scholes J, Carbonell D, Qureshi N, Xia P, Zeng L, Li S, Yu Y, Hiti AL, Yee JK, Malik P. Puthenveetil G, et al. Among authors: zeng l. Blood. 2004 Dec 1;104(12):3445-53. doi: 10.1182/blood-2004-04-1427. Epub 2004 Aug 3. Blood. 2004. PMID: 15292064
Human CD34(+) and CD34(+)CD38(-) hematopoietic progenitors in sickle cell disease differ phenotypically and functionally from normal and suggest distinct subpopulations that generate F cells.
Luck L, Zeng L, Hiti AL, Weinberg KI, Malik P. Luck L, et al. Among authors: zeng l. Exp Hematol. 2004 May;32(5):483-93. doi: 10.1016/j.exphem.2004.02.003. Exp Hematol. 2004. PMID: 15145217