ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025
Biologics-based technologies for highly efficient and targeted RNA delivery.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
What it found
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
7 citing papers in PubMed.
- Extracellular Vesicles Derived from Natural Biological Resources and Their Potential to Facilitate Skin Regeneration and Rejuvenation.Pharmaceutics · 2026Review
- Overcoming Antibiotic Resistance and Treating Bacterial Infections with Biological Nanoparticles.International journal of molecular sciences · 2025Review
- CAR Therapies: Ex Vivo and In Vivo Potential of Exosomes and Biomimetic Nanoparticles.Cancers · 2025Review
- In Vivo Behavior of Biomimetic Nanoparticles: Strategies for Clearance Avoidance, Targeting, and Functional Delivery.Molecules (Basel, Switzerland) · 2025Review
- Review
- Cryo-trojan mesenchymal stem cells as non-living tumor-homing supercarriers for enhanced drug delivery and immune activation in prostate cancer.Materials today. Bio · 2025Article
- Exosome-based nanomedicines for digestive system tumors therapy.Nanomedicine (London, England) · 2025Review
Corrections and comments
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Authors and funding
24 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The demand for RNA-based therapeutics is increasing globally. However, their use is hampered by the lack of safe and effective delivery vehicles. Here, we developed technologies for highly efficient delivery of RNA cargo into programmable extracellular vesicle-mimetic nanovesicles (EMNVs) by fabricating hybrid EMNV-liposomes (Hybs). Tissue targeting is endowed by highly efficient genetic platforms based on truncated CD63 (ΔCD63) or PTGFRN proteins. For the first time we reveal their efficiency in functionalizing EMNVs, resulting in >10-fold enhancement of nanoparticle internalization in vitro and >2-fold in vivo. RNA delivery using Hybs demonstrated efficiency of >85% in human and mouse cell lines. Comparative analysis of EMNVs and Hyb lysosome colocalization and stability suggested that Hybs enter the lysosomal compartment and escape over time, whereas EMNVs primarily avoid it. Finally, we used these technologies to generate liver-targeting Hybs loaded with therapeutic small interfering RNA and demonstrated the robust efficiency of this system in vitro and in vivo. These technologies can be adapted for manufacturing a wide range of next-generation vehicles for highly efficient, safe delivery of RNA into desired organs and tissues for therapeutic and prophylactic applications.
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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.