ArticleMolecular therapy. Methods & clinical development2015
Simple downstream process based on detergent treatment improves yield and in vivo transduction efficacy of adeno-associated virus vectors.
Article in Molecular therapy. Methods & clinical development, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 28 citations in OpenAlex.
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- Evolving membrane-associated accessory protein variants for improved adeno-associated virus production.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
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- rAAV Production and Titration at the Microscale for High-Throughput Screening.Human gene therapy · 2022Article
- Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping inMolecular therapy. Methods & clinical development · 2020Article
- A nanoparticle pseudo pathogen for rapid detection and diagnosis of virus infection.Sensors international · 2020Article
- A ligand-based system for receptor-specific delivery of proteins.Scientific reports · 2019Article
- Exon Skipping in a Dysf-Missense Mutant Mouse Model.Molecular therapy. Nucleic acids · 2018Article
- Pharmacology of Recombinant Adeno-associated Virus Production.Molecular therapy. Methods & clinical development · 2018Review
- Review
- Rapid, scalable, and low-cost purification of recombinant adeno-associated virus produced by baculovirus expression vector system.Molecular therapy. Methods & clinical development · 2016Article
Corrections and comments
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Authors and funding
6 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Recombinant adeno-associated viruses (rAAV) are promising candidates for gene therapy approaches. The last two decades were particularly fruitful in terms of processes applied in the production and purification of this type of gene transfer vectors. This rapid technological evolution led to better yields and higher levels of vector purity. Recently, some reports showed that rAAV produced by transient tri-transfection method in adherent human embryonic kidney 293 cells can be harvested directly from supernatant, leading to easier and faster purification compared to classical virus extraction from cell pellets. Here, we compare these approaches with new vector recovery method using small quantity of detergent at the initial clarification step to treat the whole transfected cell culture. Coupled with tangential flow filtration and iodixanol-based isopycnic density gradient, this new method significantly increases rAAV yields and conserves high vector purity. Moreover, this approach leads to the reduction of the total process duration. Finally, the vectors maintain their functionality, showing unexpected higher in vitro and in vivo transduction efficacies. This new development in rAAV downstream process once more demonstrates the great capacity of these vectors to easily accommodate to large panel of methods, able to furthermore ameliorate their safety, functionality, and scalability.
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Registered trials
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