Adeno-Associated Viral (AAV)
Adeno-associated viral (AAV) is a small, non-disease causing virus that can infect both dividing and non-dividing human cells. AAV cannot replicate on its own and is non-pathogenic. The AAV used for gene therapy does not insert DNA into the genome, but does deliver DNA to the host cell nucleus. Consequently, AAV vector can be used as an efficient delivery vehicle for the long-term expression of therapeutic proteins in humans.
However, because of its small size, AAV cannot be used to deliver a full length dystrophin gene or express full-length dystrophin protein. AAV is currently being utilized to deliver shortened mini/micro-dystrophin genetic sequences that will lead to expression of mini/micro-dystrophin protein in muscles throughout the body in DMD patients.