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基因治疗中的病毒载体与非病毒载体:技术发展与临床试验

Viral and non-viral vectors in gene therapy: technology development and clinical trials.

作者信息

Lundstrom Kenneth, Boulikas Teni

机构信息

Regulon Inc./BioXtal, Chemin des Croisettes 22, CH-1066, Epalinges, Swizerland.

出版信息

Technol Cancer Res Treat. 2003 Oct;2(5):471-86. doi: 10.1177/153303460300200513.

DOI:10.1177/153303460300200513
PMID:14529313
Abstract

Gene therapy as part of modern molecular medicine holds great promise for the treatment of both acute and chronic diseases and has the potential to bring a revolutionary era to cancer treatment. Gene therapy has been named the medicine of the future. For the past 10 years various viral and non-viral vectors have been engineered for improved gene and drug delivery. Although various diseases have been targeted, cancer therapy has been addressed to a large extent because of the straight forward approach. Delivery of toxic or immunostimulatory genes by viral and non-viral vectors has been investigated and encouraging results have been obtained in animal models. A large number of clinical trials have been conducted with some highly promising outcome. We propose that combinations of viruses with liposomes or polymers will solve the problem of systemic viral delivery and tumor targeting, bringing a revolution in molecular medicine and in applications of gene therapy in humans.

摘要

基因治疗作为现代分子医学的一部分,在治疗急性和慢性疾病方面具有巨大潜力,并有潜力为癌症治疗带来一个革命性的时代。基因治疗被誉为未来的医学。在过去十年中,人们设计了各种病毒和非病毒载体,以改善基因和药物递送。尽管针对了各种疾病,但由于方法直接,癌症治疗在很大程度上得到了关注。通过病毒和非病毒载体递送毒性或免疫刺激基因已得到研究,并在动物模型中取得了令人鼓舞的结果。已经进行了大量临床试验,取得了一些非常有前景的成果。我们认为,病毒与脂质体或聚合物的组合将解决全身病毒递送和肿瘤靶向问题,给分子医学以及基因治疗在人类中的应用带来一场革命。

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