Gene therapy in heart disease.
Gene therapy in heart disease.
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心脏病的基因治疗。
DOI：
10.1007/978-1-4615-1957-7_8
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发表时间：
1995
影响因子：
--
通讯作者：
Woo,SL
中科院分区：
文献类型：
--
作者：
Smith,LC;Eisensmith,RC;Woo,SL
人们之所以对基因治疗感兴趣，是因为人们意识到，对于许多人类疾病，目前对人类疾病的治疗，无论是遗传的还是其他的，通常都是针对症状或继发性缺陷，而几乎从未针对确切的生化或遗传疾病本身。理想的治疗方法可能包括实际改变或替换有缺陷的基因(Friedman，1983)。尽管外科和药学取得了显著的进步，但对5000多种遗传性疾病的治疗仍不是临床现实。最有效的药物不仅在靶器官起作用，而且在产生副作用的其他组织中起作用。此外，药物价格昂贵，必须终身服用。基因治疗是终生用药的一种选择，在没有有效药物的情况下是特殊的。
Interest in gene therapy arises from the realization that, for many human diseases, current treatment of human disease, genetic or otherwise, is generally aimed at symptoms or secondary defects and almost never at the precise biochemical or genetic disease itself. The ideal treatment could involve actual change or replacement of defective genes (Friedman, 1983). In spite of remarkable surgical and pharmaceutical advances, therapy of the more than 5000 genetic disorders is not a clinical reality. The most effective drugs act, not only in the target organ, but also in other tissues, where they produce side effects. Moreover, drugs are expensive and must be continued for lifetime. Gene therapy is an alternative to a lifetime of medication, particularity when effective drugs do not exist.