Gene Therapy Technology

Gene therapy is a technique designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. To deliver the gene into the cells, a carrier also called a vector is used. Adeno-associated viruses (AAVs) are very suited as vector, as they are derived from viruses that have the natural ability to deliver genetic material when infecting cells. For their application in gene therapy, the viruses are modified to deliver “good” genes into the cells. AAV is a small virus that is not associated to causing disease in the first place, significantly reducing the chance of a negative reaction to the vector. By now, considerable experience has been build up with AAVs for multiple applications in humans. AAVs are thus an efficient and relatively safe way to bring genes into cells. Unlike other vectors, AAVs introduce the DNA into the nucleus of the cell, but the DNA is not integrated into the cell’s own genetic material. This makes these AAV vectors very suited for use in non-dividing cells, like neurons. The new gene delivered by the vector is used by the host cell as template to produce the protein of interest, such as for instance a therapeutic antibody. Gene therapy is typically administered by injection and a very important advantage is that it needs to be given only once for a long-lasting chronic treatment.