AAV Vector Design for Gene Editing
As a leader and expert in the field of gene therapy, Creative Biolabs is able to provide state-of-art technologies to meet any requirement of our customers. We have successfully completed a series of challenges in the past years. In particular, we have established an advanced adeno-associated virus (AAV) vector-based therapeutic gene-editing platform which enables us to offer a series of high-quality services for treating a full range of diseases.
Introduction of AAV Vector Design for Gene Editing
Gene editing is a novel genetic technology that has been widely used for altering the genome of a variety of tissues or organs. In particular, the targeted DNA can be modified and inserted into specific sites of host genomes by gene editing. Pilot studies have demonstrated that nucleases play an important role in producing a series of double-strand breaks (DSBs) at specific locations of the genome. A variety of nucleases have been identified, including meganucleases, Cas9 nucleases, as well as transcription activator-like effector nucleases (TALENs). In general, the DSBs can be repaired by a number of methods, including homologous recombination, to cause desired mutations.
In recent years, gene editing has shown its potential in many research purposes, especially for gene therapy. For example, the AAV vector has been regarded as an attractive viral vector system for gene editing. Currently, a series of vectors basing on different AAV serotypes have been approved for clinical use. The data suggest that these vectors can provide a high gene transfer efficacy for many tissues, such as the brain, heart, lungs, and muscle. Furthermore, AAV vectors are capable of stimulating homology-directed repair (HDR) in the absence of a nuclease, which poses the possibility for nuclease delivery in Cas9-associated gene editing.
Figure 1. AAV Vector Design Services for Gene Editing.
Services
Genome editing technologies offer a unique opportunity of treating a range of human diseases, such as cardiovascular disease, highly penetrant disorders, HIV and Alzheimer's disease. Up to now, a wide collection of gene editing systems has been generated for recognizing site-specific genetic mutations. The effectiveness of therapeutic genome editing strategies has been evaluated in various animal models, such as mice, rats, and rabbits. Among them, the AAV vector has been considered as an effective approach for therapeutic genome editing.
As a consequence, Creative Biolabs has developed a panel of AAV vector services to provide alternative genome editing methods with a lower mutagenic risk. For instance, the expression of a factor IX cDNA has been engineered by using AAV vectors to alter the bleeding diathesis phenotype in factor IX-deficient mice models. Moreover, we also offer AAV-mediated nuclease expression services for our clients to meet the requirement of gene editing in several tissues, such as the livers and brain. In addition, the platform for assessing the immunotoxicity resulting from protein delivery has been established to guarantee the stability and safety of gene editing technologies. Now, we have developed efficient protocols for AAV vector-based gene-editing services and mainly offer two aspects of services in this field:
As one of the top CROs in the field of AAV vector development, Creative Biolabs has focused on AAV vector-based gene therapies for years and has accomplished numerous projects in this field. We can offer high-quality custom services of AAV vector design for our clients. If you are interested in our services, please 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.
