Many of the most useful biotechnology applications, from vaccines to gene therapies, require the delivery of engineered nucleic acids into human cells. The delivery vector of choice for most in vivo gene delivery applications, particularly for gene therapy, is adeno-associated virus (AAV), a small virus that can infect both dividing and quiescent murine, primate, and human cells. While AAV-based vectors effectively deliver nucleic acid cargo into cells via infection, the virus as it is deployed in humans is not known to cause disease and generally does not integrate itself into the human genome. For these reasons, it is the delivery method of choice for the two FDA-approved gene therapies on the market and an effective tool for creating isogenic animal models of disease.
Currently, challenges with AAV-based gene delivery include efficient and specific infection of the intended cell type, nucleic acid cargo size limitations, and effective targeting and/or expression of the delivered genetic cargo. This portfolio of AAV technologies from Stanford inventors preeminent in the fields of gene therapy and AAV biology represents the cutting edge of this technological domain, pushing past current limitations into the next generation of gene delivery.