Couture L A, Stinchcomb D T
Ribozyme Pharmaceuticals, Inc, Bouldar, CO 80301, USA.
Trends Genet. 1996 Dec;12(12):510-5. doi: 10.1016/s0168-9525(97)81398-4.
The ability of certain enzymatic RNA molecules, or ribozymes, to site-specifically cleave other RNA molecules opens new vistas in gene therapy. Ribozymes can be designed to target specifically a particular mRNA and inhibit protein expression, permitting 'anti-gene' therapy. Here, we describe the progress towards developing ribozymes for use in gene therapy applications. Significant advances have been made in understanding ribozyme transcription unit design and the first clinical tests of ribozyme safety in humans are soon to be initiated.
某些酶性RNA分子，即核酶，能够对其他RNA分子进行位点特异性切割，这为基因治疗开辟了新的前景。可以设计核酶使其特异性靶向特定的信使核糖核酸（mRNA）并抑制蛋白质表达，从而实现“反基因”治疗。在此，我们描述了在开发用于基因治疗的核酶方面所取得的进展。在理解核酶转录单位设计方面已取得重大进展，并且即将启动核酶在人体中的首次安全性临床试验。
