Ribozymes, DNAzymes and small interfering RNAs as therapeutics.
Ribozymes, DNAzymes and small interfering RNAs as therapeutics.
复制标题
核酶、DNAzyme 和小干扰 RNA 作为治疗剂。
作者：
M. Sioud;P. Iversen
核酸酶选择性基因沉默为研究者提供了一种新的阻断基因表达和验证药物靶点的策略。核酶、DNA酶和小干扰RNA（siRNA）正被探索作为基因表达的遗传抑制剂以及针对病毒感染、炎性病症、血液病和癌症的潜在治疗剂。我们回顾了这些分子的作用方式，特别强调它们的结构和通过特定的化学修饰来提高它们的血清半衰期的可能性。它们在细胞培养和疾病动物模型中的潜在用途也得到了强调。
Selective gene silencing by nucleic acid enzymes has provided researchers with a new strategy to block gene expression and drug target validation. Ribozymes, DNAzymes and small interfering RNAs (siRNAs) are being explored as genetic inhibitors of gene expression as well as potential therapeutics against viral infections, inflammatory disorders, haematological diseases and cancers. We review the mode of action of these molecules, with special emphasis on their construction and the possibility to enhance their serum half-lives via specific chemical modifications. Their potential use in cell cultures and in animal models for disease is also highlighted.