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Updated: Aug 15, 2026

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
Published on: September 5, 2016
Ribozymes, DNAzymes and small interfering RNAs as therapeutics
Mouldy Sioud1, Per Ole Iversen
1Institute for Cancer Research, Department of Immunology, Molecular Medicine Group, The Norwegian Radium Hospital, Montebello, 0310 Oslo, Norway. mosioud@ulrik.uio.no
Abstract:
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.
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