Anti-AAV 抗体

補足情報

This supplementary information is collated from multiple sources and compiled automatically.

The adeno-associated virus or AAV is a small virus with a simple structure known for its ability to infect human cells without causing disease. AAV belongs to the Parvoviridae family and comprises approximately 20 to 25 nanometers in size. Its simple geometry includes a capsid protein shell that encases a single-stranded DNA genome. The molecular weight of the AAV capsid is about 3.9 x 10^6 Daltons. Researchers have developed multiple serotypes such as AAV1 and AAV2 each with varying capsid structures used for different purposes. AAV effectively expresses gene delivery vehicles primarily targeting the liver muscle and central nervous system tissues. AAV ELISA and anti-AAV antibodies are critical tools for studying and quantifying these viral vectors.

Biological function summary

AAV functions as a vector for delivering genetic material into cells integrating genetic circuits or correcting genetic defects. AAV does not integrate into the host genome naturally but can establish stable long-term expression in episomal forms. AAV is known for its lack of pathogenicity making it suitable for gene therapy applications. It relies on helper viruses like adenovirus or herpes simplex virus to provide essential factors for replication. The non-pathogenic nature and the episodic persistence within host cells enable AAV-derived vectors for developing therapies and are extensively researched in the context of genetic disorders.

Pathways

AAV primarily interacts with cellular mechanisms during its entry trafficking and uncoating processes. The virus enters cells through clathrin-mediated endocytosis and relies on endosomal escape pathways for successful capsid uncoating. AAV pathways often cross with proteins like integrins which facilitate cell entry and proteasome pathways which modulate its intracellular processing. This detailed understanding of AAV's pathway interactions assists in optimizing vector design for efficient gene delivery positioning AAV as a valuable model in virology.

AAV vectors are pivotal in addressing genetic diseases like hemophilia and Leber congenital amaurosis. Hemophilia for example benefits from AAV-mediated replacement or supplement of defective clotting factor genes. In genetic eye disorders such as Leber congenital amaurosis AAV delivers functional genes to restore vision. Connections between AAV and proteins like coagulation factors in hemophilia or RPE65 related to retinal diseases highlight the virus's therapeutic potential. The ongoing research explores the role of AAV in developing innovative treatments offering hope for hereditary conditions.