AAV Design for Gene Replacement
As a world-leading service provider of the viral vector, Creative Biolabs has accumulated extensive experience from the accomplishment of different projects on adeno-associated virus (AAV) design for gene therapy, during which we have received a lot of recognition and praise from our clients. We are very proud of providing high-quality services to our valued clients to remove the difficulties of your projects.
AAV Design for Gene Replacement
Gene therapy has been considered as a powerful tool for a variety of human disease therapy, such as neurodevelopmental disorders. In particular, the central nervous system (CNS)-directed gene therapy basing on gene replacement has shown a long-term safety and efficacy in several animal models. Among them, AAV vectors are the most suitable vectors that can specifically target the CNS to provide in vivo gene transfer. Many serotypes of AAV have been identified and widely used for AAV design, ranging from AAV-2, AAV-5, and AAV-8 to AAV-9. Recent studies have shown that AAV-9 can cross the blood-brain-barrier (BBB) and has a broad distribution in the brain, which makes it a perfect virus for mediating gene therapy of the CNS. Meanwhile, the most significant progress has been made to develop gene replacement therapy basing on animal models for the treatment of various diseases. For example, a Prph2 mutation mouse has been developed to treat inherited retinal degeneration. In this study, an AAV vector carrying a Prph2 gene will be injected into the retina and the long-term efficacy has been evaluated by electron microscopy and electroretinography.
Figure 1. AAV Design Services for Gene Replacement.
Services
In gene replacement therapy, the AAV delivery system is critical to the expression of the transgene in target cells. Currently, AAV-based gene replacement strategies have been developed to treat retinal degeneration. The results indicate that AAV plays an important role in repairing retinal function and photoreceptor structure in a wide collection of animal models, such as mice, rats.
At Creative Biolabs, we offer a series of AAV design services for gene replacement and have successfully accomplished many projects in the past few years. For instance, AAV serotype 5 vectors (AAV-5) containing smCBA has been generated to transfer the Pde6b gene to the rd10 female mice. Moreover, we have established many state-of-art technologies, such as electroretinography, immunocytochemistry, western blot assay, to analyze the expression level of target genes, the safety, the function and the efficacy of AAV in gene replacement. Additionally, we are dedicated to developing helper-free human AAV systems that allow us to insert genes into a broad range of cell types with high titer and adaptability, low toxicity and immunogenicity.
Creative Biolabs is recognized as the world leader for providing the most diverse portfolio of gene therapy solutions for worldwide customers. We are always committed to assisting our clients with the most satisfactory AAV vector-based services. We have successfully established a panel of advanced technologies for exploiting a more efficient disease therapeutic regimen. For more information, please feel free to contact us or send us an inquiry.
