At CD Formulation, we recently pushed out a novel adeno-associated virus-based mRNA delivery platform. This novel platform is able to not only combine the properties of both adeno-associated viruses and mRNA but also achieve high effective mRNA delivery. We also provide highly qualified adeno-associated virus vectors and much more services utilizing mRNA delivery systems to assist our customers' biological nanoformulation research and project development.
Here, we explore and describe that the adeno-associated virus vector consists of a protein shell. This protein shell is composed of the capsid of the AAV viral vector and the AAV genome.
Fig.1 Schematic representation of AAV genomic structure. (Arvind Srivastava, et al. 2021)
Adeno-associated virus vectors, as replication-defective viruses, have great potential in mRNA delivery systems because of their significant and unique benefits as follows.
Adeno-associated virus vectors have widespread applications in vaccine therapy, protein replacement therapy, and chimeric antigen receptor T cell therapy. Recombinant adeno-associated viruses have been verified as therapeutic tools for a variety of diseases, such as ocular, neurological, metabolic, hematological, neuromuscular, cardiovascular disease, and oncology.
Fig.2 Current clinical applications of rAAV in major human diseases. (Jiang-Hui Wang, et al. 2024)
At this platform, adeno-associated virus vectors are easy to achieve tissue-specific delivery. At CD Formulation, our core team has explored devise strategies for adeno-associated virus-based mRNA delivery to achieve better transduction efficiency and tissue specificity. Our workflow of adeno-associated viral vector development includes plasmid development and production, cell expansion, plasmid transfection, viral vector production, purification, filling, and finishing.
At this stage, our workflow mainly includes the design and production of cis-plasmids, trans-plasmids, and helper plasmids.
During cell expansion, we used a HEK293-based transient transfection process for viral vector production.
The method used during our plasmid transfection is transient tri-transfection of mammalian cells or infection of mammalian cells and insect cells.
Our most common method of producing AAV viral vectors is transient transfection.
Our purification of viral particles from process- and product-related impurities includes host cell material, plasmid DNA, and empty capsids. And our viral vector purification methods include cell lysis, filtration, platform purification method, and separation of empty capsids from full capsids.
Our viral vector formulation and fill/finish processes include formulation design, concentration, and diafiltration using transient flow filtration (TFF) to final formulation and fill/finish.
Fig.3 Viral vector manufacturing process workflow, which involves various upstream, downstream, formulation, and fill/finish steps. (Arvind Srivastava, et al. 2021)
Technology: Virus-derived mRNA delivery system
Journal: Small
IF: 13.0
Published: 2024
Results:
The authors describe that the viral vectors cover adenovirus, lentivirus (LV), and adeno-associated virus. The authors have also studied the properties and applications of AAVs. They have become the main research field of viral vectors due to their advantages, such as low genotoxicity, low immunogenicity, and high delivery efficiency. From this study, the authors have found that AAV vectors are naturally non-pathogenic and replication-deficient. So AVV is used as a vector for the production of transgenic proteins, which can effectively deliver mRNA in vivo to achieve the purpose of gene therapy.
Fig.4 Virus-derived mRNA delivery system. (Menghao Yin, et al. 2024)
CD Formulation is specialized in researching our leading mRNA delivery system based on adeno-associated virus. This system includes the construction and analysis of adeno-associated virus vectors. If you have any questions about this system, please kindly contact us for in-depth discussion and communication.
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