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 AAVs(2). AAV2 is the most frequently used serotype. The most safety and efficacy data - from more than 40 completed trials - is available for this serotype as well.
for delivery to the central nervous system (CNS) AAV8 and AAV9 are being used more and more as gene therapy gains traction for CNS diseases.(9)
We offer a unique selection of Adeno-Associated Virus (AAV) antibodies for gene therapy research. Click on the links to see more product details.