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首页> 外文期刊>Journal of Biotechnology >In pursuit of new developments for gene therapy of human diseases
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In pursuit of new developments for gene therapy of human diseases

机译:追求人类疾病基因治疗的新发展

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Gene therapy aims at transferring a therapeutic gene into human somatic cells in order to treat a disease. Originally addressed to hereditary genetic disorders, gene therapy has found therapeutic applications in cancer, infectious diseases and degenerative disorders, particularly those of the nervous system. Although gene transfer into humans has been demonstrated in several clinical trials, with more than 300 currently underway worldwide, there is still no single outcome that undoubtedly showed a consistent benefit for the patient. Nevertheless, the expectations for gene therapy are still high, and the prospects of future clinical success are increasing together with the growing of the field. The development of better delivery systems specifically tailored to individual diseases, with sustained expression of the therapeutic gene in the appropriate cells, will in the end make possible true therapeutic applications of human gene transfer. (C) 1949 Elsevier Science B.V. All rights reserved.
机译:基因疗法旨在将治疗性基因转移到人体细胞中以治疗疾病。最初针对遗传性遗传疾病,基因治疗已在癌症,传染病和变性疾病(尤其是神经系统疾病)中得到治疗应用。尽管已经在数项临床试验中证明了将基因转移至人类的能力,但目前全球已有300多项研究正在进行,但仍然没有任何结果无疑显示出对患者的持续益处。然而,对基因疗法的期望仍然很高,并且随着该领域的发展,未来临床成功的前景也在增加。最终开发出专门针对个别疾病的更好的递送系统,使治疗性基因在适当的细胞中持续表达,最终将使人类基因转移的真正治疗应用成为可能。 (C)1949 Elsevier Science B.V.保留所有权利。

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