Xiaotong Fu, Wei-Chiang Chen, Christopher Argento, Peter Clarner, Vinay Bhatt, Ryan Dickerson, George Bou-Assaf, Meisam Bakhshayeshi, Xiaohui Lu, Svetlana Bergelson, John Pieracci
Recombinant adeno-associated virus (rAAV)-mediated gene therapy is a fast-evolving field in the biotechnology industry. One of the major challenges in developing a purification process for AAV gene therapy is establishing an effective yet scalable method to remove empty capsids, or viral vectors lacking the therapeutic gene, from full capsids-viral product containing the therapeutic sequence. Several analytical methods that can quantify the empty-to-full capsid ratio have been reported in the literature. However, as samples can vary widely in viral titer, buffer matrix, and the relative level of empty capsids, understanding the specifications and limitations of different analytical methods is critical to providing appropriate support to facilitate process development...
August 2019: Human Gene Therapy Methods