Rossi J J, Sarver N
Department of Molecular Genetics, Beckman Research Institute, City of Hope, Duarte, CA 91010.
Trends Biotechnol. 1990 Jul;8(7):179-83. doi: 10.1016/0167-7799(90)90169-x.
Among the landmark discoveries of recent years are ribozymes, RNA molecules which possess enzymatic, self-cleaving activities. The concept of exploiting the ribozyme catalytic center for cleaving (inactivating) a specific RNA transcript is now emerging as a potential therapeutic or preventative strategy in human diseases, veterinary medicine and agriculture. Linked to the catalytic center of the ribozyme are RNA sequences which are complementary to, and thus serve to target the ribozyme to, a unique RNA sequence. Specific association of the ribozyme with its target via base pairing, cleavage of the RNA substrate and subsequent recycling of the ribozyme make these catalytic RNA molecules attractive as antiviral agents. Theoretically, ribozymes can be adapted for the destruction of any RNA species, whatever its origin.
近年来具有里程碑意义的发现之一是核酶,即具有酶促自我切割活性的RNA分子。利用核酶催化中心切割(灭活)特定RNA转录本的概念,正作为一种潜在的治疗或预防策略出现在人类疾病、兽医学和农业领域。与核酶催化中心相连的是与独特RNA序列互补的RNA序列,因此这些序列可将核酶靶向该独特RNA序列。核酶通过碱基配对与靶标特异性结合、切割RNA底物以及随后核酶的循环利用,使得这些催化RNA分子作为抗病毒剂具有吸引力。理论上,核酶可用于破坏任何来源的RNA种类。