Adeno-associated virus (AAV) Knowledge base
Over the past decades, numerous AAV serotypes have been identified with variable tropism.
AAV establishes latency by undergoing specifically integration into a genome site, termed as the adeno-associated virus integration site 1 (AAVS1), a 4kb region on chromosome 19.
The AAV shell is approximately 25nm in diameter and encapsidates a single-stranded DNA genome of 4.7 kilobases.
AAV has been developed into a very attractive candidate for creating viral vectors for gene therapy and the creation of isogenic human disease models due to various advantages.
With the development of recombinant AAV vectors (rAAV), the adenovirus helper genes required for AAV replication have been identified, which can be cloned into plasmids for AAV production to liberate the need of helper virus. Currently, AAV production adopts the 3-plasmid co-transfection system (AAV plasmid with gene of interest, AAV packaging plasmid AAV-RC (AAV replication and AAV capsid) and pHelper (AAV helper plasmid)) in AAV-293 cells, which significantly simplifies the AAV purification process.
Though wild-type AAV is not associated with human disease, it is naturally defective and requiring helper adenovirus or herpes simplex virus (HSV) coinfection for AAV replication, so recombinant AAV (rAAV) has been an attractive vector for gene therapy.
AAV capsid engineering (AAV capsid directed evolution, AAV capsid peptide display, AAV capsid DNA shuffling)
Although more than 100 AAV serotype variants have been found in nature, significant progress has been made for rAAV production, the transduction efficiency and specificity for some tissues and cell types in animals and primates is low.
For normal tissues or organs, such as heart, liver, kidney, breast, pancreas, ovary, brain, eye, skeleton muscle, adipose tissue, etc., Genemedi systematically organizes the corresponding optimal AAV serotype, gene delivery method and injection volume for mouse and rat tissue infection.
Adeno-associated viruses (AAV) are virus particles composed of single-stranded DNA surrounded by a protein shell. Despite their simple structure, recombinant AAVs (rAAV) can perform the important role of delivering nucleic acids into cells during gene therapy. Due to the high demand for potentially curative treatments in areas of huge unmet need and the growing availability of AAV vectors, a race to launch successful gene therapy products is underway.