Can AAV integrate?
AAV vectors can integrate by homologous recombination, which is termed AAV-mediated gene targeting . In order to promote homologous recombination, a vector that contains homology arms derived from genomic chromosomal DNA that flank the genetic modification being introduced is used.
Do adenoviruses integrate into the genome?
Adenovirus vectors are generally considered to be “non-integrating” vectors because they lack native integration machinery [7, 8].
How does AAV get into the nucleus?
Studies of AAV biology reveal that AAV accumulates in the perinuclear region of cells, presumably unable traffic into the nucleus. The mechanism by which AAV enters the nucleus is largely unknown.
How does AAV work in gene therapy?
How does AAV work? Simply put, AAV is transformed from a naturally occurring virus into a delivery mechanism for gene therapy. The viral DNA is replaced with new DNA, and it becomes a precisely coded vector and is no longer considered a virus, as most of the viral components have been replaced.
Why is adenovirus used in gene therapy?
They are also used for gene therapy and as vaccines to express foreign antigens. Adenovirus vectors can be replication-defective; certain essential viral genes are deleted and replaced by a cassette that expresses a foreign therapeutic gene. Such vectors are used for gene therapy, as vaccines, and for cancer therapy.
How do non-viral vectors work in gene therapy?
The non-viral vectors are Naked DNA, particle based and chemical based. They are administered by direct administration (plasmid DNA/Naked DNA)/ chemical /physical. Most of cardiovascular clinical trials use non-viral vectors as a mode of gene transfer.
What is unusual about adeno associated virus AAV )?
AAV are not currently known to cause disease. The viruses cause a very mild immune response. Several additional features make AAV an attractive candidate for creating viral vectors for gene therapy, and for the creation of isogenic human disease models.