RNA Therapeutics - History, Design, Manufacturing, and Applications 2023
DOI: 10.5772/intechopen.107141
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Ribozymes as Therapeutic Agents against Infectious Diseases

Abstract: Ribozymes, also known as RNA enzymes, are catalytic RNA molecules capable of cleaving specific RNA sequences, leading to decreased expression of targeted genes. Recent studies suggest their role in cancer therapeutics, genetic diseases and retroviral infections. This book chapter will focus on ribozymes acting as therapeutic agents against infectious diseases caused by viral and bacterial pathogens. Firstly, we will introduce a brief history of ribozymes and a general overview of ribozymes and their characteri… Show more

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Cited by 5 publications
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“…Methods for generating multivalent aptamers have also been developed to increase affinity of the aptamer constructs for the target up to 200-fold [131]. Aptamers could theoretically also be used to deliver ribozymes and DNAzymes, single stranded nucleic acid-based enzymes that have also been explored as therapeutics, including in antiviral and anticancer applications [132,133]. A potential drawback of this approach is that both aptamers and ribozymes/DNAzymes have extensive secondary structures that are essential for function, chimeras would have to be carefully designed to prevent disruption of those structures.…”
Section: Combinatorial Approachesmentioning
confidence: 99%
“…Methods for generating multivalent aptamers have also been developed to increase affinity of the aptamer constructs for the target up to 200-fold [131]. Aptamers could theoretically also be used to deliver ribozymes and DNAzymes, single stranded nucleic acid-based enzymes that have also been explored as therapeutics, including in antiviral and anticancer applications [132,133]. A potential drawback of this approach is that both aptamers and ribozymes/DNAzymes have extensive secondary structures that are essential for function, chimeras would have to be carefully designed to prevent disruption of those structures.…”
Section: Combinatorial Approachesmentioning
confidence: 99%
“…Methods for generating multivalent aptamers have also been developed to increase the affinity of the aptamer constructs of the target up to 200-fold [ 131 ]. Aptamers could theoretically also be used to deliver ribozymes and DNAzymes, single stranded nucleic acid-based enzymes that have also been explored as therapeutics, including in antiviral and anticancer applications [ 132 , 133 ]. A potential drawback of this approach is that both aptamers and ribozymes/DNAzymes have extensive secondary structures that are essential for their function, so chimeras would have to be carefully designed to prevent the disruption of those structures.…”
Section: Future Directionsmentioning
confidence: 99%
“…Interestingly, some viral vector systems have been utilized to deliver and express ribozymes to cleave HIV RNA in humans. Some of these ribozyme-based antiviral tools are currently in various stages of human clinical trials [ 230 ]. Moreover, a recent innovation that could possibly provide additional solutions includes the generation of bacterial cells controlled by chemically synthesized genomes [ 231 ].…”
Section: Targeted Rna-cleaving Tools As Potential Antiviral Therapeuticsmentioning
confidence: 99%