Weihong Qu, Mingxi Wang, Yaqing Wu and Ruian Xu Pages 684 - 695 ( 12 )
Recombinant adeno-associated virus (rAAV) vector is one of the promising delivery tools for gene therapy. Currently, hundreds of clinical trials are performed but the major barrier for clinical application is the absence of any ideal large scale production technique to obtain sufficient and highly pure rAAV vector. The large scale production technique includes upstream and downstream processing. The upstream processing is a vector package step and the downstream processing is a vector purification step. For large scale downstream processing, the scientists need to recover rAAV from dozens of liters of cell lysate or medium, and a variety of purification strategies have been developed but not comprehensively compared till now. Consequently, this review will evaluate the scalable downstream purification strategies systematically, especially those based on the physicochemical properties of AAV virus, and attempt to find better scalable downstream strategies for rAAV vectors.
Clinical application, downstream purification, large scale, physicochemical properties, purification strategy, rAAV vector.
Yunleung Laboratory of Molecular Diagnostics, School of Medicine and Institute of Molecular Medicine, Huaqiao University, Quanzhou 362021, China.