AAVviruksia

AAVviruksia, commonly known as adeno-associated viruses, are small viruses that belong to the parvovirus family. They are not known to cause disease in humans, making them attractive candidates for gene therapy applications. AAVs are single-stranded DNA viruses that require a helper virus, such as adenovirus, to replicate efficiently. However, they can infect cells without a helper virus, entering a latent state where their genome integrates into the host cell's DNA, typically at a specific locus on chromosome 19.

The genome of AAV is about 4.7 kilobases long and encodes for proteins involved in replication, assembly, and capsid formation. The capsid, the protein shell that encloses the viral genome, is crucial for determining which cell types the virus can infect. Different AAV serotypes have distinct capsid proteins, allowing them to target various tissues and organs. This tropism is a key feature exploited in gene therapy to deliver therapeutic genes to specific cells.

Research into AAVs began in the 1960s, and their potential as gene delivery vehicles was recognized early on. Their non-pathogenic nature, low immunogenicity, and ability to infect both dividing and non-dividing cells have made them one of the most widely used viral vectors. Gene therapy trials using AAVs have shown promise in treating a range of genetic disorders, including certain forms of blindness, muscular dystrophy, and hemophilia. Challenges remain, such as the potential for immune responses against the viral capsid and the limited packaging capacity of the AAV genome. Despite these hurdles, AAVs continue to be a cornerstone of modern gene therapy research and development.