Recombinant AAVs created by the facility are based on the natural AAV2 virus.
The AAV2 genome is linear single-stranded DNA molecule of 4.7kb. The genome is organised into two open reading frames, flanked by inverted terminal repeats (ITRs) which form a hairpin structure and initiate replication. Rep is involved in DNA replication and gene expression, while Cap encodes the capsid proteins. AAVs do not replicate autonomously and a successful cycle requires the presence of a lytic helper adenovirus (Ad Helper).
|CNS||AAV1, AAV2, AAV4, AAV8, AAV9, PHP.B, PHP.eB|
|Liver||AAV7, AAV8, AAV9|
|Heart||AAV1, AAV8, AAV9|
|Lung||AAV4, AAV5, AAV6, AAV9|
|Skeletal Muscle||AAV1, AAV6, AAV7, AAV8, AAV9|
|Photoreceptors||AAV2, AAV5, AAV8|
Recombinant AAVs have manipulated so that the Rep and Cap genes are substituted with the expression cassette with the gene of interest. The ITR sequences contain the cis-acting elements necessary for replication and packaging, while the genes required from replication and assembly are provided in trans.