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Your search keyword '"Meneghini, Vasco"' showing total 4 results
Start Over Author "Meneghini, Vasco" Journal molecular therapy
4 results on '"Meneghini, Vasco"'

4. Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease

Author

Ramadier, Sophie, Chalumeau, Anne, Felix, Tristan, Othman, Nadia, Aknoun, Sherazade, Casini, Antonio, Maule, Giulia, Masson, Cecile, De Cian, Anne, Frati, Giacomo, Brusson, Megane, Concordet, Jean-Paul, Cavazzana, Marina, Cereseto, Anna, El Nemer, Wassim, Amendola, Mario, Wattellier, Benoit, Meneghini, Vasco, and Miccio, Annarita

Abstract

Sickle cell disease (SCD) is caused by a mutation in the β-globin gene leading to polymerization of the sickle hemoglobin (HbS) and deformation of red blood cells. Autologous transplantation of hematopoietic stem/progenitor cells (HSPCs) genetically modified using lentiviral vectors (LVs) to express an anti-sickling β-globin leads to some clinical benefit in SCD patients, but requires high- level transgene expression (i.e., high vector copy number [VCN]) to counteract HbS polymerization.

Published
2021
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