The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector ...
Mingchun Liu, Bruna Lima Gomes, Isabelle Mila, Eduardo Purgatto, Lázaro E.P. Peres, Pierre Frasse, Elie Maza, Mohamed Zouine, Jean-Paul Roustan, Mondher Bouzayen and Julien Pirrello Our knowledge of ...
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