Basic and clinical application of adeno-associated virus-mediated genome editing.

Traditional gene therapy (gene replacement), as the most compelling concepts in clinical medicine, have gained a breakthrough in treating inherited diseases. Adeno-associated virus (AAV) has emerged as a highly promising vector with innate ability, boosting the development of gene replacement and gene targeting. With the recent advance of engineered nucleases that work efficiently in human cells, AAV mediated-genome editing with nucleases have raised hopes for the gene therapy of inherited or non-inherited diseases. Here, we highlight applications of AAV-mediated genome editing and discuss shortcomings of AAV and nucleases that will render extension of such success in clinical gene therapy.