Pierga J Y, Cammilleri S, Benyahia B, Magdelénat H
Laboratoire de physiopathologie, institut Curie, Paris, France.
Bull Cancer. 1994 Dec;81(12):1023-42.
The development of biotechnologies offers considerable potential for new therapeutical approaches, targetting nucleic acids as information bearing molecules responsible for various pathologies. Gene therapy has, up to now, mainly aimed at compensating deficiencies of gene expression, through complex molecular and cellular constructions. Recently, an increasing interest has focused on short nucleotidic sequences (thus relatively easy to synthetize), or oligonucleotides, which could be able to specifically block the expression of mutated or overexpressed genes. When the target of these oligonucleotides is the mRNA of such genes, the strategy is called "antisense". This strategy has already led to a number of successful results in experimental models in vitro. Much more rare are actual effects in animal models and clinical trials are just being sketched. A review on the current state of the art will enlighten the physiological potential of these new molecules but also underline the still numerous issues before controlling therapeutical applications.
生物技术的发展为新的治疗方法提供了巨大潜力，将核酸作为导致各种病理状况的信息承载分子作为靶点。到目前为止，基因治疗主要旨在通过复杂的分子和细胞构建来补偿基因表达的缺陷。最近，人们越来越关注短核苷酸序列（因此相对容易合成）或寡核苷酸，它们能够特异性阻断突变或过表达基因的表达。当这些寡核苷酸的靶点是此类基因的mRNA时，该策略被称为“反义”。这一策略已经在体外实验模型中取得了许多成功的结果。在动物模型中的实际效果则更为罕见，临床试验才刚刚起步。对当前技术水平的综述将揭示这些新分子的生理潜力，但也会凸显在控制治疗应用之前仍然存在的诸多问题。
