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Ribozymes

Ribozymes

The term ribozyme is used for RNA that can act as an enzyme. Ribozymes are mainly found in selected viruses, bacteria, plant organelles, and lower eukaryotes. Ribozymes were first discovered in 1982 when Tom Cech’s laboratory observed Group I introns acting as enzymes. This was shortly followed by the discovery of another ribozyme, Ribonulcease P, by Sid Altman’s laboratory. Both Cech and Altman received the Nobel Prize in chemistry in 1989 for their work on ribozymes.
Ribozymes can be...

Ribozymes

Ribozymes

The term ribozyme is used for RNA that can act as an enzyme. Ribozymes are mainly found in selected viruses, bacteria, plant organelles, and lower eukaryotes. Ribozymes were first discovered in 1982 when Tom Cech’s laboratory observed Group I introns acting as enzymes. This was shortly followed by the discovery of another ribozyme, Ribonulcease P, by Sid Altman’s laboratory. Both Cech and Altman received the Nobel Prize in chemistry in 1989 for their work on ribozymes.
Ribozymes can be...

Riboswitches

Riboswitches

Riboswitches are non-coding mRNA domains that regulate the transcription and translation of downstream genes without the help of proteins. Riboswitches bind directly to a metabolite and can form unique stem-loop or hairpin structures in response to the amount of the metabolite present. They have two distinct regions – a metabolite-binding aptamer and an expression platform.
The aptamer has high specificity for a particular metabolite which allows riboswitches to specifically regulate...

Types of RNA

Types of RNA

Overview
Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in the regulation of gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
RNA...

RNA Interference

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...

RNA Interference

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...

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Related Experiment Video

Updated: Jul 10, 2026

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy

Published on: September 5, 2016

Therapeutic ribozymes: principles and applications.

¹Department of Molecular Biology, Beckman Research Institute of the City of Hope, Duarte, California 91010, USA. jrossi@smtplink.coh.org

Biodrugs : Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy

|November 21, 2007

Summary

Ribozymes, RNA enzymes, are emerging as therapeutic agents for diseases like HIV/AIDS. Further research into RNA metabolism and cellular mechanisms is crucial for enhancing their effectiveness in clinical applications.

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Area of Science:

Molecular Biology
RNA Therapeutics
Biochemistry

Background:

Ribozymes are RNA molecules with catalytic activity, progressing from research tools to potential therapeutic agents.
Clinical trials are underway for ribozyme-based therapies, notably against HIV, the cause of AIDS.
Improving intracellular effectiveness of ribozymes requires deeper understanding of RNA biology.

Purpose of the Study:

To review progress and experimental approaches for enhancing ribozyme intracellular effectiveness.
To survey experimental testing of potential therapeutic applications of ribozymes.
To highlight the dual role of ribozymes as research tools and therapeutic agents.

Main Methods:

Review of existing literature on ribozyme function and therapeutic applications.
Analysis of factors influencing ribozyme efficacy, including nucleic acid hybridization and cellular RNA metabolism.
Survey of experimental data from therapeutic trials and laboratory studies.

Main Results:

Ribozyme function is influenced by nucleic acid hybridization rules and cellular RNA processing mechanisms.
Knowledge of RNA metabolism and transport is key to designing more effective ribozymes.
Experimental evidence supports the potential of ribozymes in treating acquired and inherited diseases.

Conclusions:

Ribozymes show promise as therapeutic agents for various diseases, including HIV/AIDS.
Further research into RNA biology is essential for optimizing ribozyme design and delivery.
Ribozymes offer a dual utility in both elucidating gene function and treating diseases by degrading specific RNAs.