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Wild-type AAV has attracted considerable interest from gene therapy researchers due to a number of features. Chief amongst these was the virus's apparent lack of pathogenicity. It can also infect non-dividing cells and has the ability to stably integrate into the host cell genome at a specific site (designated AAVS1) in the human chromosome 19.
Self-complementary adeno-associated virus (scAAV) is a viral vector engineered from the naturally occurring adeno-associated virus (AAV) to be used as a tool for gene therapy. [1] Use of recombinant AAV (rAAV) has been successful in clinical trials addressing a variety of diseases. [ 2 ]
It was found that AAV genome inserts in less than ~10% of occasions AAV infects a cell and the expression is less than when episomally expressed. [4] AAV can only package genomes between 2 – 5.2 kb in size when they are flanked with inverted terminal repeat sequences (ITRs), but optimally holds a genome of 4.1 to 4.9 kb in length. [ 5 ]
In the 1980s, as a graduate student, he pioneered the use of AAV as a vector for therapeutic genes. [ 15 ] [ 13 ] In 1984, he cloned the virus DNA into a bacterial plasmid. This led to the recognition of the potential of AAV and also formed the foundation for the two current FDA-approved AAV gene therapies. [ 10 ]
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[1] Marines with 4th Assault Amphibian Battalion, 4th Marine Division, drive an amphibious assault vehicle off the back of the USNS SEAY, a Maritime Prepositioning Ship, at Blount Island in Jacksonville, Fla., Aug. 13, 2014. Bravo Company (-) reservists from Jacksonville, Florida, deployed to New Orleans in support of rescue and recovery ...
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