GENE-THERAPY - A NOVEL FORM OF DRUG-DELIVERY
GENE-THERAPY - A NOVEL FORM OF DRUG-DELIVERY
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DOI：
10.1056/nejm199511023331808
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发表时间：
1995-11-02
影响因子：
158.5
通讯作者：
SPRINGER, ML
中科院分区：
文献类型：
--
作者：
BLAU, HM;SPRINGER, ML
基因治疗是一种新型的药物输送方式，它利用患者细胞的合成机制来产生治疗剂。用身体来治疗自身的疾病克服了制造高度纯化蛋白质的需要。它还消除了反复静脉注射蛋白质（如血友病）或药物（如遗传性高胆固醇血症）的需要，并减少了遵守外源性药物治疗方案的困难。基因治疗的应用不仅限于罕见的遗传性疾病，而且可能扩展到常见的获得性疾病，包括癌症、心脏病和获得性免疫缺陷综合征。因此，基因治疗是可能的……
Gene therapy is a novel form of drug delivery that enlists the synthetic machinery of the patient's cells to produce a therapeutic agent. Using the body to treat its own disease overcomes the need to manufacture highly purified proteins. It also eliminates the need for repeated parenteral administration of proteins (as in hemophilia) or drugs (as in hereditary hypercholesterolemia) and reduces the difficulties of complying with exogenous-drug regimens. Applications of gene therapy are not limited to rare inherited diseases but extend potentially to common acquired disorders, including cancer, heart disease, and the acquired immunodeficiency syndrome. Gene therapy is thus likely . . .