刁勇 许瑞安. 口服重组腺相关病毒基因药物J. 药学学报, 2009,44(7): 703-709.
引用本文: 刁勇 许瑞安. 口服重组腺相关病毒基因药物J. 药学学报, 2009,44(7): 703-709.
DIAO Yong, HU Rui-An. Oral recombinant adeno-associated virus gene medicineJ. 药学学报, 2009,44(7): 703-709.
Citation: DIAO Yong, HU Rui-An. Oral recombinant adeno-associated virus gene medicineJ. 药学学报, 2009,44(7): 703-709.

口服重组腺相关病毒基因药物

Oral recombinant adeno-associated virus gene medicine

  • 摘要:

    重组腺相关病毒 (rAAV) 载体介导的口服基因药物引起业界广泛的重视。尽管经口服给药后转基因的有效表达面临许多障碍, 但该技术的有效性已得到大量实验证实。本文总结了口服rAAV基因药物的临床前研究结果, 重点阐述了该类型药物的传递、吸收、分布和基因转导等药动学特点。已证实rAAV基因药物对人体的安全性高, 但口服rAAV基因药物的临床应用仍需对其作用机制和生物药剂学特征进行深入和广泛的研究。

     

    Abstract:

    The efficacy of recombinant adeno-associated virus (rAAV) vector-mediated gene delivery to the gastrointestinal tract has been paid a considerable attention over the last 10 years, since our first report on the oral gene pill strategy in Nature Medicine, even though there are still several potential obstacles for this route to overcome in order to obtain efficient gene delivery.  The preclinical results of oral rAAV gene medicine are summarized in this review, and special attention is paid on its pharmacokinetic and pharmacodynamic aspects with an emphasis on drug delivery, absorption, distribution and transduction.  The rAAV based vectors have been shown promising results in human clinical trials with fewer safety concerns over other gene medicines.  However, the underlying mechanisms and biopharmaceutical features of oral rAAV gene medicine remain to be explored extensively and intensively to develop this novel technology as a treatment for a wider range of diseases.

     

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