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

22 May 2019

Abstract excerpt

Liver-directed gene therapy for the coagulation disorder hemophilia showed safe and effective results in clinical trials using adeno-associated viral vectors to replace a functional coagulation factor, although some unmet needs remain. Lentiviral vectors (LVs) may address some of these hurdles because of their potential for stable expression and the low prevalence of preexisting viral immunity in humans. However,...

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PMID: 31118293
DOI: 10.1126/scitranslmed.aav7325

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