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Gene therapy is an innovative approach to treating a disease by introducing a functional gene or inactivating/editing a disease-causing gene. To do this, gene therapy uses modified noninfectious viruses called “vectors” that can enter certain cells. The transfer of a working gene into those cells helps produce a functional protein.
Gene therapies work through a variety of approaches including functional gene transfer. Functional genes are usually delivered into the cells of the body by inserting them into an inactivated viral shell (the vector), which carries the gene to specific target cells. The gene can be delivered directly to the person (in vivo gene therapy) or into cells that have been taken from the person, then treated, then returned to the person (ex vivo gene therapy).
The gene therapies for hemophilia B that are currently being studied in clinical trials use a certain type of vector called an adeno-associated virus, or AAV.
AAV-based gene therapy is built on trusted scientific advances and years of proven clinical research, creating breakthrough treatments for a variety of diseases over the past decade—two with FDA/EMA approvals—and more than 250 clinical trials currently underway.
Watch Dr. Pipe discuss why AAV vectors are used in gene therapy.
AAV-based gene therapy uses nonpathogenic, nonreplicating viruses and consists of a capsid (vector) encasing a therapeutic gene (transgene). Through its design, it facilitates the accurate and efficient activation of the transgene inside its targeted destination, for example, the liver.
There are many different types of AAV vectors that can target specific areas of the body. Some AAV vectors are more precise in their targets, while others target a wider, less specific range of cells and tissues.
AAV vectors also have lower levels of immunogenicity compared with other viral vectors such as retroviruses, lentiviruses, and adenoviruses.
For liver-directed AAV gene therapy, serotypes AAV2, AAV5, AAV6, AAV8, and AAV9 have been shown to exhibit liver tropism, with some exhibiting narrower tropism than others.
Watch Dr. Pipe discuss how targeted delivery of a gene is achieved using AAV vectors.
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