Adeno-Associated Virus is a protein shell surrounding and protecting a small, single-stranded DNA genome of approximately 4.8 kilobases. AAV belongs to the parvovirus family and is dependent on co-infection with other viruses, mainly adenoviruses, in order to replicate.(1) AAV vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to the growth of the gene therapy field.(2)
AAVs are the most commonly used method for delivering gene-editing tools like CRISPR-Cas9. The Use of AAVs in gene-replacement therapy approved by the FDA for patients suffering from spinal muscular atrophy and congential blindness. The advantages of AAV are reduced risk of genomic integration, broad tissue targeting possibilities, clinically manageable immunogenicity. Current challenges are low genetic "cargo load" and reduced efficiency when using second AAV vector to increase "cargo load", as well as undesired off-target effects due to long-term expression of gene-editing molecules and scalability issues regarding AAV manufacturing.(8)
Below you will find an overview of some important target organs for AAV(2). We offer a variety of hand-picked antibodies and proteins for your research. By clicking on the links you will be directed to the available products.
We offer a unique selection of Adeno-Associated Virus (AAV) antibodies for gene therapy research. Click on the links to see more product details.