Basic principles of technology
What are the key elements of gene therapy technology?
What are the key elements of gene therapy technology?
AAVs are commonly used in the study of potential gene therapies because their protein shell can be repurposed to deliver a therapeutic
gene cassette
to target cells. AAVs do not cause disease and are unable to replicate in the target cell without co-infection of a helper virus.1
1
Therapeutic gene cassette
The therapeutic gene cassette contains a functional gene and a promoter to instruct the cell to make protein or knock down protein. Long-lasting gene expression depends, in part, on the type of promoter used to drive its expression.2 Tissue-specific promoters are advantageous because of their ability to direct expression of protein in target cells, while having no effect in other cell types.3,4
2
AAV vector delivery system
An AAV-based protein shell is engineered to deliver gene cassettes to target cells.
AAV vectors have shown long-lasting gene expression after administration of a single treatment in preclinical and clinical studies.
3
Administration technology
There are a variety of administration technologies that have been shown to deliver the relevant functional gene into target cells. Delivery strategies can be systemic, such as via intravenous infusion, or local, such as into the brain or the eye.5
See below for an animated demonstration of how gene transfer technology functions within the body:
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