At present,recombinant adeno-associated virus is the most commonly used vectors for in vivo gene therapy products.For producing virus vectors,plasmids are believed to be the important starting raw material.The designing and managing of plasmids directly contribute to the biological mechanisms,pharmaceutical properties,efficacy,and safety of products.Therefore,understanding the properties of plasmids and their gene elements is the basis for the"quality by design"of such products.The functions of different plasmids are introduced in this paper,the construction of main genetic elements are summarized,the considerations for designing of plasmids are discussed,the compliance for developing and manufacturing of plasmids are reviewed,in order to provide some references for the development and management of gene therapy products,especially for the recombinant adeno-associated virus products.
维普
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