ArticleMolecular therapy : the journal of the American Society of Gene Therapy2013
Genetically engineered microvesicles carrying suicide mRNA/protein inhibit schwannoma tumor growth.
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 185 papers.
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Who cites it
185 citing papers in PubMed, 334 citations in OpenAlex.
- Extracellular vesicles for next-gen therapeutics and drug delivery.Molecular biomedicine · 2026Review
- Metabolic Regulation of Immune Responses: Molecular Mechanisms, Diseases, and Therapeutic Targets.MedComm · 2026Review
- Endogenous Engineering Reprograms Extracellular Vesicles for Enhanced Therapeutic Function.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Review
- Bioengineered extracellular vesicles escape lysosomal degradation and deliver Tet-PKM2 for macrophage immunometabolic reprogramming and periodontitis treatment.Bioactive materials · 2026Article
- Distinguishing Pseudotransduction and True Transduction Enables Characterization and Bioengineering of Extracellular Vesicle-Adeno-Associated Virus Vectors.Journal of extracellular vesicles · 2026Article
- Extracellular vesicles in spine tumors: biological roles, immune modulation, and therapeutic implications.European spine journal : official publication of the European Spine Society, the European Spinal Deformity Society, and the European Section of the Cervical Spine Research Society · 2026Review
- Engineering the Future: Strategic Advances in Extracellular Vesicle-Mediated Drug Delivery Systems.International journal of nanomedicine · 2026Review
- Exosomes as Emerging Nanocarriers for Targeted Cancer Therapy.International journal of nanomedicine · 2026Review
- Extracellular vesicles in viral disease management.Current research in microbial sciences · 2026Review
- Extracellular vesicle-based therapeutic strategies for spinal tumors and associated nerve damage: advances, challenges, and future directions.Frontiers in cell and developmental biology · 2026Review
- In Vitro and Animal Studies of Human Natural Killer Cell-Derived Exosomes for the Treatment of Otitis Media.Neuroscience bulletin · 2025Article
- Extracellular vesicles as biomarkers and drug delivery systems for tumor.Acta pharmaceutica Sinica. B · 2025Review
- Extracellular vesicles as missiles for enhanced anti-tumor efficacy of oncolytic viruses: from disseminating oncolysis and anti-tumor immunity to targeted delivery.Cell communication and signaling : CCS · 2025Review
- Gene therapy and genome-editing for schwannoma in NF2-related schwannomatosis: current understanding and future directions.Journal of neuro-oncology · 2025Review
- Exosome-powered neuropharmaceutics: unlocking the blood-brain barrier for next-gen therapies.Journal of nanobiotechnology · 2025Review
- Control of Physical and Biochemical Parameters Influencing Exogeneous Cargo Protein Association to Extracellular Vesicles Using Lipid Anchors Enables High Loading and Effective Intracellular Delivery.Journal of extracellular biology · 2025Article
- Exosomes: from basic research to clinical diagnostic and therapeutic applications in cancer.Cellular oncology (Dordrecht, Netherlands) · 2025Review
- Relationship between exosomes and cancer: formation, diagnosis, and treatment.International journal of biological sciences · 2025Review
- The biology of exosomes and exosomal non-coding RNAs in cardiovascular diseases.Frontiers in pharmacology · 2025Review
- Delivering therapeutic RNA into the brain using extracellular vesicles.Molecular therapy. Nucleic acids · 2024Review
125 more citing papers are in PubMed but not listed here.
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Authors and funding
9 authors at 3 institutions in 2 countries.
Funding
Abstract
Microvesicles (MVs) play an important role in intercellular communication by carrying mRNAs, microRNAs (miRNAs), non-coding RNAs, proteins, and DNA from cell to cell. To our knowledge, this is the first report of delivery of a therapeutic mRNA/protein via MVs for treatment of cancer. We first generated genetically engineered MVs by expressing high levels of the suicide gene mRNA and protein-cytosine deaminase (CD) fused to uracil phosphoribosyltransferase (UPRT) in MV donor cells. MVs were isolated from these cells and used to treat pre-established nerve sheath tumors (schwannomas) in an orthotopic mouse model. We demonstrated that MV-mediated delivery of CD-UPRT mRNA/protein by direct injection into schwannomas led to regression of these tumors upon systemic treatment with the prodrug (5-fluorocytosine (5-FC)), which is converted within tumor cells to 5-fluorouracil (5-FU)-an anticancer agent. Taken together, these studies suggest that MVs can serve as novel cell-derived "liposomes" to effectively deliver therapeutic mRNA/proteins to treatment of diseases.
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