Abstract：OBJECTIVE To summarize the structural design strategy and safety assessments of rAAV packaging systems. METHODS Based on the research progress and international review experience of commercial rAAV products, the design strategy and safety assessments of rAAV packaging systems were summarized. The design strategy and safety assessments of rAAV packaging systems were summarized. RESULTS The scientific evaluation of the pharmaceutical design of rAAV for human should cover multiple aspects, because various types of AAVs differ in tissue selectivity, viral assembly, transgene expression. Meanwhile, the packaging system is diverse, and the production process is complex. CONCLUSION The considerations referring to the rAAV structure and mechanisms of producing replication-competent AAV are proposed, and we hope to improve the communications between developers and regulators.
卢加琪, 韦薇. 对重组腺相关病毒基因治疗产品包装体系和分子设计的思考[J]. 中国药学杂志, 2020, 55(16): 1386-1393.
LU Jia-qi, WEI Wei. Some Thinkings on the Structural Design and Packaging System of Raav-Based Gene Therapy Products. Chinese Pharmaceutical Journal, 2020, 55(16): 1386-1393.
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