Adeno‐associated viral vectors: depend(o)ble stability

Abstract

Recombinant adeno‐associated viral (rAAV) vectors can mediate the safe and long‐term correction of genetic diseases in animal models following a single administration, and have been shown to persist and are safe in humans following delivery to lung, sinus, skeletal muscle, brain, and liver. The physiochemical properties of rAAV vector virions are amenable to industry‐accepted manufacturing methodologies, long‐term storage, and direct in vivo administration.