Toxicity and Safety Determination of AAV Vector Service
As one of the top CROs in the field of gene therapy, Creative Biolabs has focused on the development of AAV vector-based gene therapies for years and has successfully accomplished numerous challenging projects in this field. We can offer a range of high-quality adeno-associated virus (AAV) vector construction, safety profiling, toxicity evaluation and animal modeling services for our clients.
Introduction of AAV Vector
AAV vectors have been considered as a leading gene delivery platform and have been widely used for gene therapy in a wide variety of human diseases, especially for malignant tumors. Previous studies have revealed that AAV vectors can effectively bind to targeted cells and be expressed in the cell nucleus with a low or no toxicity. Most AAV vectors are designed by using the AAV-2 serotype. These AAV-2 vectors can successfully transduce a number of tissues in humans, such as liver, lung, as well as the brain. In recent years, the efficiency of AAV-2 transduction has been evaluated by many organizations and companies, and the data suggest that viral trafficking and second-strand synthesis play an important role in regulating gene expression of AAV in disease models. In addition, several novel AAV vector strategies have been developed for improving the safety of AAV vectors, for instance, all viral coding sequences of AAVs have been removed to reduce the toxicity, attain more stable expression levels in specific cells.
Figure 1. Converting a virus into a vector. (Thomas, 2003)
Services
Significant improvements have been made in gene therapy, and AAV vector-based therapy has shown promising results in treating various types of diseases. Pilot studies have demonstrated that some AAV vectors can enter host cells to help the process of replication, but the expression of AAV genes can cause the toxicity of in vivo animal models. As a consequence, a number of technologies have been established to evaluate the safety and toxicity of AAV vectors in pre-clinical studies. As an expert in AAV-based gene therapy, Creative Biolabs works on a full range of safety profiling and toxicity assessment of AAV vector services to promote the progress of gene therapy in human diseases. In our labs, pseudotyping AAV2 vector genome with capsids from other kinds of AAV serotypes has shown more efficient and safer gene transfer in treating melanoma. Moreover, we also offer a panel of AAV vector administration assays, such as arterial blood gas analysis, inflammatory cytokines analysis, as well as lung mechanics analysis, to detect the safety of AAV vectors. In recent studies, a single-dose administration AAV-CFTR vector has been evaluated by our labs. The results suggest that this vector plays a key role in mediating a stable and safe CFTR gene transfer in rabbit models. Additionally, we have developed a number of in vivo immunogenicity platforms to determine cellular immune responses against a model antigen expressed by AAVs.
Figure 2. Toxicity and Safety Determination of AAV Vector Service.
As a leading service provider in gene therapy for disease treatment, Creative Biolabs now provides professional virus vector services to support the growing interest in this filed. Nowadays, we provide a variety of AAV vectors services focused on designing, optimization, safety, and toxicity evaluation. If you are interested in our services, please feel free to contact us for closer communication to learn how we can be involved in your project. Separate services or integrated end-to-end solutions are all welcomed.
Reference
- Thomas, C. E.; et al. (2003). Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet. 4(5): 346-58.
