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
-
Mesenchymal Stem/Stromal Cells: A New "Cells as Drugs" Paradigm. Efficacy and Critical Aspects in Cell Therapy
Current Pharmaceutical Design Inhibitors of the Proteolytic Activity of Urokinase Type Plasminogen Activator
Current Pharmaceutical Design Cervical Cancer Diagnosis: Insights into Biochemical Biomarkers and Imaging Techniques
Combinatorial Chemistry & High Throughput Screening Preparation and <I>In Vitro/Vivo</I> Evaluation of Folate-conjugated Pluronic F87-PLGA/TPGS Mixed Nanoparticles for Targeted Drug Delivery
Current Drug Delivery Lipid-based Nanoplatforms in Cancer Therapy: Recent Advances and Applications
Current Cancer Drug Targets Patent Selections
Recent Patents on Inflammation & Allergy Drug Discovery Antitumor Therapeutic Strategies Based on the Targeting of Epidermal Growth Factor-Induced Survival Pathways
Current Drug Targets Hyaluronic Acid/Parecoxib-Loaded PLGA Microspheres for Therapy of Temporomandibular Disorders
Current Drug Delivery MicroRNA Targeting as a Therapeutic Strategy Against Glioma
Current Molecular Medicine Prospects of Non-Coding Elements in Genomic DNA Based Gene Therapy
Current Gene Therapy Ultrasound-Mediated Cancer Therapeutics Delivery using Micelles and Liposomes: A Review
Recent Patents on Anti-Cancer Drug Discovery Recent Patents on Proteasome Inhibitors of Natural Origin
Recent Patents on Anti-Cancer Drug Discovery Let-7a Could Serve as A Biomarker for Chemo-Responsiveness to Docetaxel in Gastric Cancer
Anti-Cancer Agents in Medicinal Chemistry Serum miRNAs Signature Plays an Important Role in Keloid Disease
Current Molecular Medicine Encountering and Advancing Through Antiangiogenesis Therapy for Gliomas
Current Pharmaceutical Design Environmentally Sensitive Paramagnetic and Diamagnetic Contrast Agents for Nuclear Magnetic Resonance Imaging and Spectroscopy
Current Topics in Medicinal Chemistry Mechanisms of Drug Resistance to Vascular Endothelial Growth Factor (VEGF) Inhibitors
Anti-Cancer Agents in Medicinal Chemistry Challenges in the Design of Clinically Useful Brain-targeted Drug Nanocarriers
Current Medicinal Chemistry Comparing the Interaction of Cyclophosphamide Monohydrate to Human Serum Albumin as Opposed to Holo-Transferrin by Spectroscopic and Molecular Modeling Methods: Evidence for Allocating the Binding Site
Protein & Peptide Letters Recent Advances of MEK Inhibitors and Their Clinical Progress
Current Topics in Medicinal Chemistry