ArticleJournal of extracellular vesicles2025
Efficient Scaling up EV-AAVs Production via Cellular Nanoporation for Familial Hypercholesterolaemia Therapy.
Article in Journal of extracellular vesicles, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
- Efficient Scaling up EV-AAVs Production via Cellular Nanoporation for Familial Hypercholesterolaemia Therapy.Journal of extracellular vesicles · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
21 authors.
Funding
Abstract
Adeno-associated virus (AAV)-mediated gene therapies face critical clinical limitations, including immune-mediated neutralization by pre-existing antibodies and dose-dependent hepatotoxicity. Extracellular vesicle-encapsulated AAVs (EV-AAVs) offer a promising solution by shielding AAVs from antibody recognition, yet existing production methods remain inefficient and impractical for clinical application. Here, we developed a cellular nanoporation (CNP) platform that enables scalable, high-yield generation of EV-AAVs, achieving an approximately 11-fold increase in production efficiency compared with conventional methods. In LDLR-deficient murine models with pre-existing neutralizing antibodies (1:200), EV-AAV-LDLR at half the standard AAV dose robustly restored hepatic LDL receptor expression and attenuated atherosclerosis progression. Notably, EV-AAV exhibited superior immune evasion capabilities, maintaining 2.3-fold higher hepatic transduction efficiency than conventional AAV upon secondary dosing due to antibody shielding. Importantly, EV-AAV therapy markedly reduced hepatotoxicity, with serum AST/ALT levels comparable to saline-treated controls, thereby overcoming a critical safety barrier of high-dose AAV treatment. These results demonstrate CNP as a clinically translatable platform for scalable EV-AAV manufacturing, enabling effective multi-dose regimens while overcoming key immunological and toxicity barriers in liver-directed gene therapy for familial hypercholesterolaemia.
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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.