Ribozyme gene therapy: applications for molecular medicine.
RNA enzymes--ribozymes--are being developed as treatments for a variety of diseases ranging from inborn metabolic disorders to viral infections and acquired diseases such as cancer. Ribozymes can be used both to downregulate and to repair pathogenic genes. In some instances, short-term exogenous delivery of stabilized RNA is desirable, but many treatments will require viral-mediated delivery to provide long-term expression of the therapeutic catalyst. Current gene therapy applications employ variations on naturally occurring ribozymes, but in vitro selection has provided new RNA and DNA catalysts, and research on trans-splicing and RNase P has suggested ways to harness the endogenous ribozymes of the cell for therapeutic purposes.
Lewin
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Dept of Molecular Genetics and Microbiology and the Powell Gene Therapy Center, University of Florida, Gainesville 32610-0266, USA. lewin@mgm.ufl.edu
Hauswirth
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D000163:Acquired Immunodeficiency Syndrome / Q000628:therapy; D000483:Alleles; D000818:Animals; D001483:Base Sequence; D003062:Codon; D004260:DNA Repair; D000229:Dependovirus / Q000235:genetics; D015536:Down-Regulation; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D007438:Introns; D008954:Models, Biological; D008969:Molecular Sequence Data; D009369:Neoplasms / Q000628:therapy; D016337:RNA, Catalytic / Q000235:genetics* / Q000627:therapeutic use*; D014161:Transduction, Genetic
D003062:Codon; D016337:RNA, Catalytic