While there are many approaches to treating Duchenne with gene therapy, the strategy that is most prevalent in Duchenne is gene replacement with a micro-dystrophin.

Even though adeno-associated viruses (AAVs) used for gene therapy are not known to cause disease in humans, your body will still recognize the virus as a foreign particle and mount an immune response.

The term pre-existing antibodies refers to a person who has been exposed to AAV at some point in their life and is now producing antibodies. If a person has a high number of antibodies to AAV they are typically ineligible for receiving a therapy that uses that AAV.

Transgenes are carried into cells using vectors. In the case of current Duchenne gene therapy programs, a virus called Adeno-associated virus (AAV) is used to deliver the transgene into the cells.