Central Nervous System Disease
Adeno-associated virus (AAV) vectors have become powerful tools in the gene therapies for central nervous system (CNS) diseases. Aided by first-class technologies and experienced scientists, Creative Biolabs is more than happy to offer professional AAV vector design services for CNS diseases' therapy.
AAV Vector-based Gene Therapy
AAV is a type of small single-stranded DNA nonenveloped virus belonging to the genus Dependoparvovirus and the family Parvoviridae. Despite it is infectious to humans and some primate species, AAV exhibits quite a low immunogenicity and pathogenicity with the ability to infect both dividing and nondividing cells. When infecting host cells, AAVs show different tropisms and infect a wide range of cell types usually in a manner of integration into the genome of the infected cells. All these characteristics make AAVs considerable candidates for gene therapy applications.
As a gene delivery vector, AAV displays some similarities and advantages over adenoviral vectors and lentiviral vectors, such as high transduction efficiency, long-term expression, non-pathogenicity, as well as limitation of small carrying capacity (only 5 kb). However, the development of recombinant AAV pushes it to be one of the most prospective and extensively used gene transfer tools. Currently, AAV vectors-based gene delivery has been widely applied for various challenging diseases' therapies clinically, such as CNS disease, inherited diseases, tumors, etc.
Figure 1. Adeno-associated virus (AAV) biology and variant generation. (Kotterman, 2014)
AAV Vector Design for CNS Diseases Therapy
CNS is the most important part of the human nervous system consisting of the brain and spinal cord. The principal functions of CNS are to transmit, store and process information, producing various psychological activities and controlling behaviors. Once the structure or function of CNS is affected, a diversity of CNS disorders will occur. The blood-brain barrier makes it challenging for conventional CNS disease treatment to achieve an ideal therapeutic effect. In recent years, immunotherapy and gene therapy have brought good news to patients with CNS diseases.
AAV vector-based gene delivery has made great progress in CNS disease therapy and exhibited great advantages, such as long-term gene expression and no apparent toxicity. There are several recombinant AAV vectors with different capsid proteins and tropisms have been developed, among which AAV serotype 9 (AAV9) is the preferred vector for CNS-directed gene therapy. Different from previously utilized CNS gene transfer AAV vectors (i.e., AAV2, AAV5, and AAV8), AAV9 is able to easily cross the blood-brain barrier and targets the neurons and astrocytes in the brain and spinal cord, suggesting a great clinical therapeutic value for CNS treatment.
Figure 2. Routes of gene delivery to the CNS. (Bowers, 2011)
As a specialist in the gene therapy field, Creative Biolabs provides both diverse recombinant AAV products and customized services for a series of CNS diseases' gene therapies, which are mainly listed as follows:
AAV has become one of the most promising and widely used gene delivery systems. Aiming to provide the most comprehensive gene therapy services with the highest quality, Creative Biolabs has successfully established an advanced gene therapy platform to offer global clients with custom AAV products and services that best fit your needs.
What you need to do is just directly contact us or send us an inquiry.
References
- Kotterman, M.A.; Schaffer, D.V. (2014). Engineering adeno-associated viruses for clinical gene therapy. Nature Reviews. Genetics. 15(7): 445-451.
- Bowers, W.J.; et al. (2011). Genetic therapy for the nervous system. Human Molecular Genetics. 20(R1): R28-R41.
