Abstract
Gene Therapy Gene therapy using adeno-associated virus (AAV) vectors has shown safety and efficacy in hemophilia. However, AAV vectors have limitations hindering their efficacy in some patients. The use of lentiviral vectors (LVs) has been explored as a possible alternative; however, preclinical data reported low transduction efficacy, possibly owing to fast clearance by phagocytes. Milani et al. developed a shielded LV able to escape phagocytosis by expressing the phagocytosis inhibitor CD47 on its surface. Upon intravenous administration in monkeys, the LVs showed high transduction efficacy without signs of toxicity. Thus, LV-mediated gene therapy might be an effective strategy for treating hemophilia. Sci. Transl. Med. 11 , eaav7325 (2019).
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