Abstract
Ribozymes are RNA molecules that have the ability to catalyse the cleavage and formation of covalent bonds in RNA strands at specific sites. The “hammerhead” motif, approximately 30-nucleotide long, is the smallest endonucleolytic cis-acting ribozyme structure found in natural circular RNAs of some plant viroids. Hammerhead ribozymes became appealing when it was shown that it is possible to produce trans-acting ribozymes directed against RNA sequences of interest. Since then, gene-tailored ribozymes have been designed, produced and given to cells to knock down the expression of specific genes. At present, this technology has advanced so much that many hammerhead ribozymes are being used in clinical trials. With this work we would provide some guidelines to design efficient trans-acting hammerhead ribozymes as well as review the recent results obtained with them as gene therapy tools.
Keywords: hammerhead ribozymes, gene knockdown, gene therapy, gene drugs, synthetic oligonucleotides
Current Gene Therapy
Title: Synthetic Hammerhead Ribozymes as Therapeutic Tools to Control Disease Genes
Volume: 5 Issue: 1
Author(s): L. Citti and G. Rainaldi
Affiliation:
Keywords: hammerhead ribozymes, gene knockdown, gene therapy, gene drugs, synthetic oligonucleotides
Abstract: Ribozymes are RNA molecules that have the ability to catalyse the cleavage and formation of covalent bonds in RNA strands at specific sites. The “hammerhead” motif, approximately 30-nucleotide long, is the smallest endonucleolytic cis-acting ribozyme structure found in natural circular RNAs of some plant viroids. Hammerhead ribozymes became appealing when it was shown that it is possible to produce trans-acting ribozymes directed against RNA sequences of interest. Since then, gene-tailored ribozymes have been designed, produced and given to cells to knock down the expression of specific genes. At present, this technology has advanced so much that many hammerhead ribozymes are being used in clinical trials. With this work we would provide some guidelines to design efficient trans-acting hammerhead ribozymes as well as review the recent results obtained with them as gene therapy tools.
Export Options
About this article
Cite this article as:
Citti L. and Rainaldi G., Synthetic Hammerhead Ribozymes as Therapeutic Tools to Control Disease Genes, Current Gene Therapy 2005; 5 (1) . https://dx.doi.org/10.2174/1566523052997541
DOI https://dx.doi.org/10.2174/1566523052997541 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
Call for Papers in Thematic Issues
Programmed Cell Death Genes in Oncology: Pioneering Therapeutic and Diagnostic Frontiers (BMS-CGT-2024-HT-45)
Programmed Cell Death (PCD) is recognized as a pivotal biological mechanism with far-reaching effects in the realm of cancer therapy. This complex process encompasses a variety of cell death modalities, including apoptosis, autophagic cell death, pyroptosis, and ferroptosis, each of which contributes to the intricate landscape of cancer development and ...read more
Related Journals
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
- Announcements
Related Articles
-
Circulating Tumor Stem Cells as Biomarkers for Cancer Progression
Recent Patents on Biomarkers Editorial (Thematic Issue: Implication of Non-coding RNAs in Cancer Biology and Cellular Physiology)
Current Genomics Current Issues in the Utility of Blood Oxygen Level Dependent MRI for the Assessment of Modulations in Tumor Oxygenation
Current Medical Imaging CD44 and EpCAM: Cancer-Initiating Cell Markers
Current Molecular Medicine Targeted Ultrasound Imaging of Cancer: An Emerging Technology on its Way to Clinics
Current Pharmaceutical Design Recent Progress in the Development of ATP-Competitive and Allosteric Akt Kinase Inhibitors
Current Topics in Medicinal Chemistry Kinase Inhibitors Targeting Anti-angiogenesis as Anti-cancer Therapies
Current Angiogenesis (Discontinued) A Unique Intracellular, Extracellular and Transmembrane Circulation of Potassium Ions in the Auditory Inner Ear as an Anticarcinogenic Principle? Part 1
Inflammation & Allergy - Drug Targets (Discontinued) Biodistribution and Pharmacokinetics of I-131 Labelled 4- Iodophenylacetic Acid
Current Radiopharmaceuticals Role of Natural Plant Products Against Alzheimer’s Disease
CNS & Neurological Disorders - Drug Targets Alkaloids from Cyanobacteria with Diverse Powerful Bioactivities
Mini-Reviews in Medicinal Chemistry Schizophrenia: A Systematic Review of the Disease State, Current Therapeutics and their Molecular Mechanisms of Action
Current Medicinal Chemistry Connecting Parkinsons Disease and Drug Addiction: Common Players Reveal Unexpected Disease Connections and Novel Therapeutic Approaches
Current Pharmaceutical Design Recent Advances in the Imaging of Programmed Cell Death
Current Pharmaceutical Design Nanomedicine to Overcome Cancer Multidrug Resistance
Current Drug Metabolism Nanotechnology Platforms; An Innovative Approach to Brain Tumor Therapy
Medicinal Chemistry Novel Phospholipid-Based Labrasol Nanomicelles Loaded Flavonoids for Oral Delivery with Enhanced Penetration and Anti-Brain Tumor Efficiency
Current Drug Delivery Preparation and Biodistribution of Technetium-99m-Labeled Bis- Misonidazole (MISO) as an Imaging Agent for Tumour Hypoxia
Medicinal Chemistry Natural Monophenols as Therapeutics, Antioxidants and Toxins; Electron Transfer, Radicals and Oxidative Stress
The Natural Products Journal Signaling Intermediates (MAPK and PI3K) as Therapeutic Targets in NSCLC
Current Pharmaceutical Design