About AAV vectors
Adeno-associated virus (AAV) is a naturally occurring non-pathogen virus. In gene therapy, the AAV’s have most of their own DNA removed and replaced with selected promoters and other functional elements in addition to the desired transgene sequences. The resulting so-called AAV vector, which unlike its natural counterpart is incapable of replication, is then used to deliver the transgene into the cells of the targeted tissue of the patient.
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