ABSTRACT
Adeno-associated virus (AAV) identification and understanding have emerged as a new delivery vector for gene therapy. The ability to generate recombinant AAVs that are devoid of viral genes and carry a DNA sequence of interest for therapeutic application has been established as a safe approach for gene therapies. It has also led to several regulatory approvals. Even though limitations of viral vector renders its application to challenging human diseases, new approaches of vector engineering and gene cassette design are aiding in overcoming these barriers.
