Research ArticleGene Therapy

Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates

See allHide authors and affiliations

Science Translational Medicine  22 May 2019:
Vol. 11, Issue 493, eaav7325
DOI: 10.1126/scitranslmed.aav7325

You are currently viewing the editor's summary.

View Full Text

Log in to view the full text

Log in through your institution

Log in through your institution

Vectors in stealth mode

Gene therapy using adeno-associated viral vectors (AAVs) has shown safety and efficacy in patients with hemophilia. However, AAVs have limitations hindering their efficacy in a subgroup of patients. The use of lentiviral vectors (LVs) has been explored as possible alternative; however, preclinical data reported low transduction efficacy possibly due to fast clearance by phagocytes. Now, Milani et al. developed a shielded LV able to escape phagocytosis by increasing the content of the phagocytosis inhibitor CD47 on their surface. Upon intravenous administration in monkeys, the LVs showed high transduction efficacy without signs of toxicity. The results suggest that LV-mediated gene therapy might be an effective strategy for treating hemophilia and possibly other disorders.

View Full Text