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Chinese Journal of Clinical Pharmacology and Therapeutics ›› 2010, Vol. 15 ›› Issue (5): 577-583.

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Immunity and strategy to recombinant adeno-associated virus vector

LU Chao1,2, WAN Jiang-bo1,2, TANG Ming-qing1,2, XIAO Wei-dong2,3, XU Rui-an1,2   

  1. 1Molecular Medicine Engineering Research Center, Ministry of Education, 2Institute of Molecular Medicine, Huaqiao University, Quanzhou 362021,Fujian, China;
    3Department of Pediatrics, University of Pennsylvania,Philadelphia 19104, Pennsylvania, USA
  • Received:2010-01-11 Revised:2010-05-07 Online:2010-05-26 Published:2020-09-16

Abstract: Recombinant adeno-associated viral vectors (rAAV) have been demonstrated as a significant promising viral vector for human gene therapy based on its high safety and good targeting activity in preclinical tests and clinical trials, and there are 71 clinical trials have dong or been doing. Part of them have gain an excellent success in the mant with Hemophilia, retinal disease, Parkinson's disease, cystic fibrosis, cancer et al. With the success in clinical trials, people process a series extensive and deeply research with the immunity induced by rAAV and the responding strategy respond to. In this review we systematically analyze the immunity caused by capsid and transgene product. Apart from this, some strategies, partly based on our work, to eliminate the immunity caused by capsid such as change the capsid, administration route, and eliminate the expression of endo-capsid are reviewed as well.

Key words: Recombinant adeno-associated viral vectors, Immunity, Capsid, Transgene product, Endo-capsid

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