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Inside "Custom Cars” For Gene Delivery

Gene therapy shows great promise to be potentially transformational treatments for a number of single-gene disorders, such as Duchenne muscular dystrophy, hemophilia and cystic fibrosis.

Gene therapy uses an engineered vector to deliver a replacement copy of a gene to diseased cells. In the case of single-gene disorders, introducing a working gene can help restore function of a faulty or missing protein. 

While it’s a straightforward concept, it can be difficult to execute. Successful vectors must be able to carry the gene, which sometimes can be very large; target the right type of cell for a specific disease; insert the new genetic material into the cell; and “activate” the gene to create the missing protein. Scientists also need to engineer vectors to avoid an immune response that could influence how well the therapy works.

In recent years, vectors derived from adeno-associated virus (AAV) have become among the most commonly used molecular delivery vehicles in gene therapy. This viral agent is a popular source of vectors because it doesn’t make people sick, does not integrate into the host genome, and is able to deliver genetic material to a variety of cell types. 

Read on to learn about the key features that go into designing an AAV vector.