Anti-AAV9 antibody [HL2370] - BSA and Azide free

Supplementary info

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

Activity summary

Adeno-associated virus serotype 9 (AAV9) is a viral vector used in gene therapy known for its ability to transduce cells by delivering genetic material. Popular as a genetic tool AAV9 is utilized because of its efficient transfection capabilities in a variety of tissues. It consists of a protein capsid which forms an icosahedral structure important for its ability to encapsulate and deliver therapeutic DNA. The AAV9 capsid has an approximate molecular mass of 60 kilodaltons per subunit assembling into a larger capsid particle. AAV9 exhibits a broad expression profile being able to transduce diverse tissues including muscle liver central nervous system and cardiac tissues.

Biological function summary

AAV9 targets enable the efficient delivery of genes by trafficking across the cellular barriers leading to gene expression within target cells. Part of the larger family of adeno-associated viruses AAV9's specificity for different tissues allows researchers to target certain organs for therapeutic purposes. The AAV9 capsid engages with cell surface receptors and undergoes internalization by endocytosis. Interestingly different serotypes within this family vary slightly in tissue tropism and selection of the appropriate serotype affects therapeutic outcomes.

Pathways

AAV9 interacts with cellular regulatory mechanisms involved in transporting genetic material to the nucleus where it integrates into the genome or exists episomally. It is notably applied in pathways related to muscle and CNS diseases where AAV9 enhances the delivery of therapeutic genes. It operates in conjunction with certain cellular proteins that facilitate endosomal escape and nuclear entry making it effective for addressing genetic defects linked with these pathways.

Associated diseases and disorders

AAV9 has been linked to therapeutic strategies for conditions such as spinal muscular atrophy (SMA) and certain types of muscular dystrophy. Utilization of AAV9 allows for the delivery of corrective genes potentially reversing or halting disease progression. In SMA for example AAV9 is used to deliver survival motor neuron (SMN) gene which is deficient in patients. Similarly in muscular dystrophy AAV9 aids in the administration of dystrophin or microdystrophin genes which play an important role in muscle integrity and function.