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An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype.
Weber L, Poletti V, Magrin E, Antoniani C, Martin S, Bayard C, Sadek H, Felix T, Meneghini V, Antoniou MN, El-Nemer W, Mavilio F, Cavazzana M, Andre-Schmutz I, Miccio A. Weber L, et al. Among authors: antoniou mn. Mol Ther Methods Clin Dev. 2018 Aug 4;10:268-280. doi: 10.1016/j.omtm.2018.07.012. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30140714 Free PMC article.
The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity.
Montiel-Equihua CA, Zhang L, Knight S, Saadeh H, Scholz S, Carmo M, Alonso-Ferrero ME, Blundell MP, Monkeviciute A, Schulz R, Collins M, Takeuchi Y, Schmidt M, Fairbanks L, Antoniou M, Thrasher AJ, Gaspar HB. Montiel-Equihua CA, et al. Mol Ther. 2012 Jul;20(7):1400-9. doi: 10.1038/mt.2012.50. Epub 2012 Mar 20. Mol Ther. 2012. PMID: 22434141 Free PMC article.
138 results