AAV Design for Gene Addition
As one of the current most popular therapies, gene therapy has been shown to be effective in treating a variety of disease types. Creative Biolabs provides a series of adeno-associated viruses (AAV) design services for our clients all over the world. Equipped with a team of professional scientists, we are capable of providing you with a wide spectrum of customized services on gene addition. We can accommodate the specific properties of your project and provide flexible integrated solutions.
AAV Design for Gene Addition
AAV has become one of the most popular genes transfer systems in a wide variety of pre-clinical or clinical studies. Previous studies have indicated that the AAV vector is equipped with all properties for vector design, such as the broad cellular tropism, strong interaction with anti-AAV antibodies, as well as high transduction efficiency. As a result, a number of strategies have been developed by scientists to design AAV vectors for various types of disease therapy, especially for cancer gene therapy. In general, the standard procedure for AAV design is based on appropriately inverted terminal repeats (ITRs), which regulate the replication and packaging of AAV in vector producer cells. Meanwhile, the recombinant AAV vector has also been generated by using a promoter driving gene of interest and a poly(A) tail. Additionally, efficient gene addition and expression can also be achieved by using AAV vectors.
However, the random insertion may lead to the attenuation or complete silencing of expression in host cells. In recent years, genomic safe harbors (GSHs) have been generated to avoid unwanted transgene expression and reduce the risk of the host genome. Moreover, scientists have revealed that clustered regularly interspaced short palindromic repeats (CRISPR)-associated molecular cloning techniques are widely used for inserting target genes in specific sites.
Figure 1. AAV Design Services for Gene Addition.
Services
Gene therapy has proven its efficacy and safety in a selection of pre-clinical or clinical trials. Among them, most gene therapy strategies developed for therapeutic applications depend on viral vector-based gene addition. Currently, Creative Biolabs offers a wide collection of AAV design services to improve the efficacy of gene addition and expression in different kinds of cells. We have established a series of platforms to integrate genetic materials into target sites of host cells. For instance, our novel DNA double-strand breaks (DSBs) platform provides an attractive opportunity for precisely gene addition at a specific genomic location. In recent studies, the ability of RNA-guided Cas9- and meganucleases-mediated delivery systems to insert genes has been compared in our labs. Meanwhile, several factors have been identified to associate with the effectiveness of these gene addition platforms, including the accessibility and the frequency of repetitive sequences of target sites. In addition, we also offer a number of AAV vector-based gene addition services to insert potential therapeutic payloads into many cell types.
Figure 2. Genomic structure of AAV and AAV vectors. (Santiago-Ortiz, 2016)
With the commitment of being your best AAV design partner, Creative Biolabs has established the utmost efficient integrated solutions to innovate and accelerate your gene therapy projects for various types of diseases, especially for malignant tumors. We have won a good reputation among our worldwide customers for successfully accomplishing numerous challenging projects in this field. We have experienced experts and advanced platforms that are able to provide excellent services. If you are interested in our services, please feel free to contact us to get more information and a detailed quote for your project.
Reference
- Santiago-Ortiz, J.L.; et al. (2016). Adeno-associated virus (AAV) vectors in cancer gene therapy. J Control Release. 240: 287-301.
