Single Gene Delivery
Adeno-associated virus (AAV) has been treated as a favorite viral tool for both basic research and clinical applications. In addition to gene therapy, AAV can also be used to transiently express genes of interest in a variety of cell types. With years of experience, Creative Biolabs is able to offer AAV vector design service for gene expression. Particularly, we are capable of designing AAV vectors for single gene delivery to study the potential function of a novel gene.
AAV Vector for Single Gene Delivery
During the process of gene therapy, the introduction of therapeutic genes into target cells, leading to an efficient and stable expression of the transgene with the minimal adverse effect is of great importance. Viral vectors are ideal vectors for gene transfer as they can easily enter cells and deliver their genetic material into the nucleus. During the past, the non-pathogenic and persistent long-term nature of AAV infection, combined with its wide range of infectivity, made this virus an important candidate as a therapeutic gene transfer vector. In addition, AAV vectors have a number of important advantages over other vectors which make them suitable for gene delivery. For example, all viral genes can be deleted from recombinant AAV vectors, thereby yielding an efficient transgene delivery vehicle with enhanced safety and reduced immunogenicity. Furthermore, AAV vectors are also capable of infecting a wide range of cell types, such as muscle, liver, brain, lung, retina, heart, and pancreas cells.
Figure 1. Adeno-associated virus (AAV) vectors. (Zacchigna, 2014)
Despite their advantageous properties, AAV vectors can and must be improved in numerous ways to enhance their utility from the lab to the clinic. To date, vectors based on AAV2 (rAAV2) are the most studied vectors used in clinical trials for numerous diseases including cystic fibrosis, hemophilia B, prostate and melanoma cancers, Canavan disease, Alzheimer's disease, Parkinson's disease, muscular dystrophy, rheumatoid arthritis, and HIV. What's more, various efforts to apply rational or combinatorial rAAV design strategies have been developed to extend rAAV delivery to previously refractory cell types.
Services
As a leading company in the gene therapy field, Creative Biolabs has focused on advances in designing AAV vectors to change tropism, enhance delivery efficiency, and evade antibody neutralization. Particularly, we have successfully established top technology platforms for AAV vector design and construction. Our professional scientists are able to generate novel AAV vector for transduction of specific cells by numerous approaches, including but not limited to the mosaic plasmid, directed evolution, receptor targeting via chemical or genetic engineering, as well as random peptide library display.
If you are interested in the service we offer, please feel free to contact us for more information.
Reference
- Zacchigna, S.; et al. (2014). Adeno-associated virus vectors as therapeutic and investigational tools in the cardiovascular system. Circulation research. 114(11), pp.1827-1846.
