Anti-gene therapy: the use of ribozymes to inhibit gene function.
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.
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Ribozyme Pharmaceuticals, Inc, Bouldar, CO 80301, USA.
Stinchcomb
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D001483:Base Sequence; D005786:Gene Expression Regulation / Q000187:drug effects*; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D008969:Molecular Sequence Data; D009690:Nucleic Acid Conformation; D012323:RNA Processing, Post-Transcriptional; D016337:RNA, Catalytic / Q000737:chemistry / Q000494:pharmacology*; D014158:Transcription, Genetic
D016337:RNA, Catalytic