ArticleMolecular therapy : the journal of the American Society of Gene Therapy2013
Displaying high-affinity ligands on adeno-associated viral vectors enables tumor cell-specific and safe gene transfer.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 101 papers.
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Who cites it
101 citing papers in PubMed, 179 citations in OpenAlex.
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- Bipolar CD4-targeted dual-DARPin-55/57 lipid nanoparticle enables efficient CRISPR/Cas-mediated HIV-1 DNA excision and reactivation blockade in latent CD4 T cell lines.Materials today. Bio · 2026Article
- An Expanded Toolbox for Versatile Chemical Editing of Adeno-Associated Virus.Angewandte Chemie (International ed. in English) · 2026Article
- AAV capsid sites breakdown: large protein insertions impact on vector dynamics.Frontiers in bioengineering and biotechnology · 2026Article
- Antibody-guided AAV vectors for antigen-specific delivery of suicide genes.Gene therapy · 2026Article
- Delivery platforms forFrontiers in immunology · 2026Review
- Intelligent design of tumor microenvironment-responsive Adeno-associated virus vectors: overcoming delivery barriers and enabling precision therapy.Medical oncology (Northwood, London, England) · 2025Review
- A facile chemical strategy to synthesize precise AAV-protein conjugates for targeted gene delivery.Molecular therapy. Oncology · 2025Article
- In vivo CAR cell therapy: from bench to bedside.Journal of hematology & oncology · 2025Review
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- GFP-on mouse model for interrogation of in vivo gene editing.Nature communications · 2025Article
- Tumor-specific AAV delivery of interleukin-12 enhances antitumor immunity and safety in ovarian cancer xenograft mouse model.Molecular therapy. Oncology · 2025Article
- Enhanced conversion of T cells into CAR T cells by modulation of the MAPK/ERK pathway.Cell reports. Medicine · 2025Article
- Shaping viral immunotherapy towards cancer-targeted immunological cell death.Frontiers in oncology · 2025Review
- αFAP-specific nanobodies mediate a highly precise retargeting of modified AAV2 capsids thereby enabling specific transduction of tumor tissues.Molecular therapy. Methods & clinical development · 2024Article
- T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Targeted gene delivery systems for T-cell engineering.Cellular oncology (Dordrecht, Netherlands) · 2024Review
- Viral and nonviral nanocarriers forNano research · 2024Article
41 more citing papers are in PubMed but not listed here.
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Authors and funding
7 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene transfer vectors derived from the adeno-associated virus (AAV) have recently received increasing attention due to substantial therapeutic benefit in several clinical trials. Nevertheless, their great potential for in vivo gene therapy can only be partially exploited owing to their broad tropism. Current cell surface targeting strategies expanded vector tropism towards transduction of cell types that are inefficiently infected naturally, but failed to restrict or fully re-direct AAV's tropism. Hypothesizing that this limitation can be overcome by equipping natural receptor-blinded AAV vectors with high-affinity ligands, we displayed designed ankyrin repeat proteins (DARPin) as VP2 fusion proteins on AAV capsids ablated for natural primary receptor binding. These second generation targeting vectors demonstrated an as of yet unachieved efficiency to discriminate between target and non-target cells in mono- and mixed cultures. Moreover, DARPin-AAV vectors delivered a suicide gene precisely to tumor tissue and substantially reduced tumor growth without causing fatal liver toxicity. The latter caused death in animals treated with conventional AAV vectors with unmodified capsids, which accumulated in liver tissue and failed to affect tumor growth. This novel targeting platform will be key to translational approaches requiring restricted and cell type-specific in vivo gene delivery.
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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.