Adeno associated virus
- Adeno-Associated Virus
Adeno-associated virus (AAV) is a small virus that infects humans and some other primate species. It is a member of the Parvoviridae family and is notable for its ability to integrate into the host genome, making it a valuable tool in gene therapy.
Structure and Genome[edit | edit source]
AAV is a non-enveloped virus with a single-stranded DNA genome. The genome is approximately 4.7 kilobases in length and contains two open reading frames (ORFs): rep and cap. The rep gene encodes proteins necessary for viral replication, while the cap gene encodes the capsid proteins that form the virus's protective shell.
Life Cycle[edit | edit source]
AAV requires a helper virus, such as an adenovirus or herpesvirus, to replicate. In the absence of a helper virus, AAV can integrate into the host cell's genome, typically at a specific site on chromosome 19 in humans. This integration is non-pathogenic and does not cause disease, which is why AAV is considered a safe vector for gene therapy.
Applications in Gene Therapy[edit | edit source]
AAV vectors are widely used in gene therapy due to their ability to deliver genetic material to a variety of cell types with high efficiency and low immunogenicity. AAV vectors have been used in clinical trials for the treatment of genetic disorders such as hemophilia, cystic fibrosis, and muscular dystrophy.
Advantages and Limitations[edit | edit source]
AAV vectors have several advantages, including their ability to infect both dividing and non-dividing cells, long-term expression of the transgene, and low risk of insertional mutagenesis. However, they have a limited packaging capacity, which restricts the size of the therapeutic gene that can be delivered.
Research and Development[edit | edit source]
Ongoing research is focused on improving the efficiency and specificity of AAV vectors, as well as expanding their use to treat a wider range of diseases. Advances in CRISPR technology and other genome editing tools are also being integrated with AAV vectors to enhance their therapeutic potential.
Also see[edit | edit source]
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