Gene therapy has become a treatment method for many diseases. Adeno-associated virus (AAV) is one of the most common virus vectors, is also widely used in the gene therapy field. During the past two decades, the retrograde axonal transportability of AAV has been discovered and utilized. Many studies have worked on the retrograde axonal transportability of AAV, and more and more people are interested in this field. This review describes the current application, influence factors, and mechanism of retrograde axonal transportability of AAV and predicted its potential use in disease treatment in near future.