Dal Palù C, Palù G, Rossi G P
Dipartimento di Medicina Clinica e Sperimentale, Università degli Studi di Padova.
Ann Ital Med Int. 1996 Oct;11 Suppl 2:55S-59S.
Since the discovery of DNA and the genetic code the main goals of molecular medicine have been: to understand the genetic basis of diseases, and to develop strategies capable of providing long-term cure. In the last 2 decades methodological progress has made it technically feasible to pursue both these goals. In its classical conception gene therapy consists in the introduction of genes into somatic cells in order to correct a specific genetic defect or to provide cells with a new function which will ultimately result to be advantageous for the patient. These strategies have been proposed for the treatment of numerous diseases, both hereditary, mono- or polygenic, and acquired in nature. The purpose of this review is to discuss the problems inherent in gene therapy, currently pursued strategies and future clinical perspectives.
自从发现DNA和遗传密码以来,分子医学的主要目标一直是:了解疾病的遗传基础,并制定能够实现长期治愈的策略。在过去20年中,方法学上的进展使得追求这两个目标在技术上变得可行。按照其经典概念,基因治疗在于将基因导入体细胞,以纠正特定的遗传缺陷或赋予细胞一种新功能,最终对患者有利。这些策略已被提出用于治疗多种疾病,包括遗传性的单基因或多基因疾病以及后天性疾病。本综述的目的是讨论基因治疗中固有的问题、目前所采用的策略以及未来的临床前景。