ArticleScience translational medicine2019
Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates.
Article in Science translational medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 85 papers.
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Who cites it
85 citing papers in PubMed.
- Systemic delivery of phagocytosis-shielded retroviral vectors enables in vivo HSC gene therapy for sickle cell disease.Cell stem cell · 2026Article
- Pharmacokinetic and pharmacodynamic characterization of CD8-targeted lentiviral vector forMolecular therapy. Advances · 2026Article
- Assessment of F/HN-pseudotyped lentiviral vector following intravenous delivery to mice.Gene therapy · 2026Article
- Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome models.Nature communications · 2026Article
- Low-density lipoproteins in human serum competitively inhibit the binding and entry of vesicular stomatitis virus.Molecular therapy. Advances · 2026Article
- Gene therapy for liver diseases: methods, challenges and opportunities.Journal of nanobiotechnology · 2026Review
- In vivo CAR T cell generation using retargeted and functionalized lentiviral vectors with reduced immunogenicity.Nature communications · 2026Article
- Transforming Hemophilia Management: Lessons from Gene Therapy Clinical Trials.Molecular biotechnology · 2026Review
- Normothermic perfusion of human livers for profiling lentiviral vector pharmacokinetics and transduction.Molecular therapy. Advances · 2026Article
- Lipid droplets are rigid and physically suppress phagocytosis, unless cell compression or stretching activates actomyosin.Molecular biology of the cell · 2026Article
- In Vivo CAR-T Therapies-A New Era of Programmable Immunity.International journal of molecular sciences · 2026Review
- FromImmune network · 2026Review
- Article
- Collagen Type II-Targeting Lentiviral Gene Therapy for Mucopolysaccharidosis IVA.Current issues in molecular biology · 2025Article
- Targeted Hepatic Delivery of Bioactive Molecules via Nanovesicles: Recent Developments and Emerging Directions.Journal of personalized medicine · 2025Review
- Gene Therapy of Beta Hemoglobinopathies.Biomedicines · 2025Review
- In vivo gene editing of human hematopoietic stem and progenitor cells using envelope-engineered virus-like particles.Nature biotechnology · 2025Article
- Spatiotemporal liver dynamics shape hepatocellular heterogeneity and impact in vivo gene engineering.Journal of hepatology · 2025Article
- In vivo CAR cell therapy: from bench to bedside.Journal of hematology & oncology · 2025Review
- Review
25 more citing papers are in PubMed but not listed here.
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Authors and funding
20 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
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, systemic LV administration to hemophilic dogs was associated to mild acute toxicity and low efficacy at the administered doses. Here, exploiting intravital microscopy and LV surface engineering, we report a major role of the human phagocytosis inhibitor CD47, incorporated into LV cell membrane, in protecting LVs from uptake by professional phagocytes and innate immune sensing, thus favoring biodistribution to hepatocytes after systemic administration. By enforcing high CD47 surface content, we generated phagocytosis-shielded LVs which, upon intravenous administration to nonhuman primates, showed selective liver and spleen targeting and enhanced hepatocyte gene transfer compared to parental LV, reaching supraphysiological activity of human coagulation factor IX, the protein encoded by the transgene, without signs of toxicity or clonal expansion of transduced cells.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.