PNA——肽核酸。用作基因治疗药物]。

Nordisk medicin Pub Date : 1998-10-01
P E Nielsen
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引用次数: 0

摘要

简要介绍了基因治疗“抗原”和“反义”药物的原理。这些药物的目标是细胞的遗传物质——要么是DNA本身,要么是它的信使分子mRNA。通过利用碱基互补原理,“抗原”,特别是“反义”药物可以很容易地设计针对特定基因的药物,抑制其活性在医学上是有利的。这些可能是病毒或致癌基因。重点讨论了利用肽核酸(PNA)作为基因治疗药物的前景。PNA是一种合成的结构DNA模拟物,在化学上与肽和蛋白质的关系更密切,迄今为止在开发基因治疗药物和诊断工具方面显示出非常有前途的化学和(分子)生物学特性。
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[PNA--peptide nuclei acids. Used as gene-therapeutic drugs].

The principles of gene therapeutic "antigen" and "antisense" drugs are briefly presented. The target of such drugs is the genetic material of the cell--either the DNA itself, or its messenger molecules, mRNA. By exploiting base-complementary principles, "antigen" and especially "antisense" drugs can easily be designed to target specific genes, the activity of which it is medically advantageous to inhibit. These could be virus- or oncogenes. In particular, the prospects of using peptide nucleic acids (PNA) as gene therapeutic drugs are discussed. PNA is a synthetic structural DNA mimic, which chemically is more closely related to peptides and proteins and which has so far shown very promising chemical and (molecular) biological properties in terms of its development into gene therapeutic drugs as well as diagnostic tools.

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