Genes are the blueprints of the human body. These DNA-based messages provide the body’s cells with instructions on how to make proteins that are critical to its function. The expression of faulty proteins is often caused by corrupted or missing genes, or by erroneous modifications to downstream products of genes.
Traditional pharmaceutical-based treatments typically modify components of the cell or its environment to induce a therapeutic response. Gene therapy, however, is focused on utilizing viral-based technologies that deliver therapeutic genes to human cells or tissues to correct the genetic defect directly.
Harnessing viruses, otherwise known as vectors, with their naturally evolved capabilities to deliver therapeutic genetic material to living cells, is one of the most exciting advances in modern medicine, having broad future therapeutic implications for a vast array of diseases. Historically, there are several recombinant viral vectors available for the delivery of therapeutic genetic cargo to the body’s cells. Recombinant adeno-associated virus (AAV) has become the dominant gene-delivery vector of choice, aimed at curing or treating disease by delivering new or replacement genes to code for therapeutic proteins in humans. AAV offers the advantages of no pathogenicity, efficient long-term gene expression, ease of genetic manipulation, and the property of low, or in many cases, absent immune response. No other gene delivery tool offers such a desirable array of properties.