Why are AAVs used in gene therapy?
AAV, or adeno-associated virus, is currently the main viral vector that researchers use and further develop for gene therapy because it is considered to be non-pathogenic to humans and because it has been successfully altered to prevent its integration into the genome, thus eliminating DNA damage and unpredictable …
What is AAV used for?
The AAV vector is then used to deliver normal copies of genes to the right tissues or organs in the body, but it now delivers the therapy that has been engineered into it. Today, AAV technology has advanced to target a wide range of tissues and cells for the treatment of many genetic diseases.
What is a nonviral vector?
Non-viral vectors are DNA plasmids that can be delivered to the target cells as naked DNA or in association with different compounds such as liposomes, gelatin or polyamine nanospheres.
How does AAV infect cells?
When AAV infects a cell alone, its gene expression is repressed (AAV does not replicate), and its genome is incorporated into the host genome (into human chromosome 19). In rare cases, lysis can occur without a helper virus, but usually AAV can not replicate and kill a cell on its own.
Why is AAV a good vector?
One of the features of AAV is its ability to specifically integrate to establish latent infection. Current AAV vectors do not have this ability, and the development of such a vector would ensure long-term transgene expression in tissues without problems associated with insertional mutagenesis.
What is aav9 vector?
AAV vectors. AAVs are small viruses (25 nm) of the Parvoviridae family belonging to the Dependovirus genus, which is indicative of their dependence on a helper virus for productive infection [52]. These viruses have a nonenveloped capsid that stores genetic material in the form of a single-stranded DNA [52].
Is AAV a retrovirus?
Overall, AAV can be produced at a moderately high titer and can infect target cells efficiently, but less so than adenovirus. Retrovirus (e.g. MMLV) can introduce genetic material into the genome of the host cell, making it great for long-term stable expression.
What is the difference between viral and nonviral vectors?
There are also different types of AAVs that can infect different kinds of cells. Because of this, AAVs are widely used in gene therapy today to target different diseases based on the types of organ and tissues they affect.
What is ex vivo gene therapy?
Ex vivo gene therapy involves the genetic modification of cells outside of the body to produce therapeutic factors and their subsequent transplantation back into patients. Various cell types can be genetically engineered.
What is Moi in virology?
Multiplicity of infection (MOI) is a frequently used term in virology which refers to the number of virions that are added per cell during infection. If one million virions are added to one million cells, the MOI is one.