Research Advances on Increasing the Transduction Efficiency of Recombinant Adeno-associated Viral Vectors

doi:10.13560/j.cnki.biotech.bull.1985.2015.09.007

Abstract

Abstract: The recombinant adeno-associated virus(rAAV)vector has emerged as one of the promising and commonly-used vectors in gene therapy research. The first gene therapy drug in clinic approved in Europe is based on rAAV. Due to the relatively limited transduction efficiency, the cost of the rAAV-mediated treatment is expensive. Additionally, high dose of rAAV may trigger host immune response, resulting in the curative effect reduced. Therefore, how to enhance the transduction efficiency of rAAV has been a hot issue in gene therapy. Hitherto there are five common methods to achieve this goal：selecting tissue-specific tropism serotypes and variants, using proteasome inhibitors, mutating capsid surface-exposed amino-acids, increasing second-strand DNA synthesis, and producing self-complementary vectors. In this paper we systemically review the above methods from the aspects of principle, status of application, advantages and disadvantages.

Key words: recombinant adeno-associated virus vector, gene therapy, transduction efficiency

Yin Zifei, Wang Li’na, Wang Yuan, Ling Chen. Research Advances on Increasing the Transduction Efficiency of Recombinant Adeno-associated Viral Vectors[J]. Biotechnology Bulletin, 2015, 31(9): 49-59.

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