Gene therapies consist of a recombinant nucleic acid intended for the modulation or manipulation of a genetic sequence. A single administration of a novel gene therapy has the potential to be curative, with a durable long-term beneﬁt to patients. Adeno-associated viral vectors (AAVs) have become the viral vector of choice for in vivo delivery of therapeutic transgenes as they are mildly immunogenic, can effectively transduce a variety of human tissues and cells, and have low levels of genomic integration. Central to the effective translation of data generated in discovery studies to the clinic is the selection of appropriate animal species for pivotal non-clinical studies.
This review, conducted by our non-clinical support team, aims to support the selection of appropriate animal models for nonclinical studies to advance the development of novel adeno-associated virus gene therapies.