Engineering of Targeted Viral Gene Delivery Vehicles
Based on extensive expertise in viral structure-function relationships, our company is dedicated to providing customers with optimization services of viral gene delivery vehicles to generate viral vectors with novel gene delivery capabilities, thus meeting multiple gene delivery needs in the treatment of rare diseases. Our protein engineering approach includes vector targeting by pseudotyping, vector targeting using adaptors, and genetic incorporation of targeting ligands.
The failure of target genes to be expressed effectively in target cells is currently the greatest difficulty in gene therapy clinical trials, and improving the targeting of viral vectors is one way to address this problem. The complexity of viruses, such as complex multimeric protein shells or capsids, presents a challenge in designing viral vectors with new properties, including resistance to immune responses and enhanced targeting ability. The rational design of targeted viral gene delivery vehicles for gene therapy has yielded successful advances in vivo and in vitro to a certain extent.
Several major protein engineering technologies, such as the pseudotyping of virus vectors, have been used in virus vector engineering to achieve effective delivery of exogenous genes, which is expected to expand the utility of engineering vectors. These approaches have shown great promise in vitro, and future in vivo analysis, especially for targeting approaches and solving concerns about the immunogenicity of vectors. With a better understanding of viral genomics and rare disease pathology, more effective and safe targeted gene therapy regimens will be developed and are expected to be applied to the clinical practice of rare disease treatment.
Fig. 1 Overview of rational protein engineering strategies for viral vectors. (Schaffer D V, et al., 2008)
We provide customers with a wide range of services for the optimization of viral vectors for gene delivery, with the aim of designing and producing viral vectors with customized, user-defined gene delivery characteristics. We have developed a variety of techniques for targeting viral vectors to specific cells, including systems that use proteins from other viruses, systems that use adaptors to couple targeting ligands to vectors, and systems that genetically integrate targeting moiety into the viral genome.
We use viral envelope proteins from another virus to produce viral vectors to help our customers restrict or broaden the host cell range to meet the needs of gene delivery in gene therapies for rare diseases.
We are committed to helping customers build adaptor-based viral vector systems, which are selective and flexible targeting systems that restrict gene transfer to the cell type of interest, accelerating the development of targeted gene therapies for rare diseases.
We provide customers with genetic incorporation of targeting ligands including peptide ligands and small peptide motifs as well as library screening and ablation of native vector tropism to achieve the desired targeting specificity of viral vectors.
Our company focuses on protein engineering technologies for viral vector engineering to provide customers with the optimization of vectors such as adenovirus vectors, AAV vectors, and retrovirus/lentivirus vectors for effective gene delivery in gene therapies of rare diseases. If you are interested in our services, please feel free to contact us for more details.
- Schaffer, D. V.; et al. Molecular engineering of viral gene delivery vehicles. Annu. Rev. Biomed. Eng., 2008, 10: 169-194.
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