Abstract
To date, the general assumption was that most mutations interested protein-coding genes only. Thus, only few illustrations have mentioned here that mutations may occur in non-protein coding genes such as microRNAs (miRNAs). We thus report progress in delineating their contribution as phenotypic modulators, genetic switches and fine-tuners of gene expression. We reasoned that browsing their contribution to genetic disease may provide a framework for understanding the proper requirements to devise miRNA-based therapy strategies, in particular the relief of an appropriate dosage. Gain and loss of function of miRNA enforce the need to respectively antagonize or supply the miRNAs. We further categorized human disease according to the different ways in which the miRNA was altered arising either de novo, or inherited whether as a mendelian or as an epistatic trait, uncovering its role in epigenetics. We discuss how improving our knowledge on the contribution of miRNAs to genetic disease may be beneficial to devise appropriate gene therapy strategies.
Keywords: anti-miRs, disease, dosage, genetics, genotype, microRNA, mimics.
Current Gene Therapy
Title:MicroRNAs in Genetic Disease: Rethinking the Dosage
Volume: 12 Issue: 4
Author(s): Alexandra Henrion-Caude, Muriel Girard and Jeanne Amiel
Affiliation:
Keywords: anti-miRs, disease, dosage, genetics, genotype, microRNA, mimics.
Abstract: To date, the general assumption was that most mutations interested protein-coding genes only. Thus, only few illustrations have mentioned here that mutations may occur in non-protein coding genes such as microRNAs (miRNAs). We thus report progress in delineating their contribution as phenotypic modulators, genetic switches and fine-tuners of gene expression. We reasoned that browsing their contribution to genetic disease may provide a framework for understanding the proper requirements to devise miRNA-based therapy strategies, in particular the relief of an appropriate dosage. Gain and loss of function of miRNA enforce the need to respectively antagonize or supply the miRNAs. We further categorized human disease according to the different ways in which the miRNA was altered arising either de novo, or inherited whether as a mendelian or as an epistatic trait, uncovering its role in epigenetics. We discuss how improving our knowledge on the contribution of miRNAs to genetic disease may be beneficial to devise appropriate gene therapy strategies.
Export Options
About this article
Cite this article as:
Henrion-Caude Alexandra, Girard Muriel and Amiel Jeanne, MicroRNAs in Genetic Disease: Rethinking the Dosage, Current Gene Therapy 2012; 12 (4) . https://dx.doi.org/10.2174/156652312802083602
DOI https://dx.doi.org/10.2174/156652312802083602 |
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
-
Lithium and Kidney, 60 Years Later
Current Drug Safety The Discovery and Development of Selective Estrogen Receptor Modulators (SERMs) for Clinical Practice
Current Clinical Pharmacology Epigenetics in Vascular Disease – Therapeutic Potential of New Agents
Current Vascular Pharmacology Editorial (Thematic Issue: Calcium in Heart Disease: The Ubiquitous Ion)
Medicinal Chemistry Pegylated Liposomal Doxorubicin in Salvage Chemotherapy for Multiple Myeloma Patients
Current Cancer Therapy Reviews Autophagy: For Better or for Worse, in Good Times or in Bad Times …
Current Molecular Medicine Pathomechanisms of Myocardial Dysfunction in Sepsis
Endocrine, Metabolic & Immune Disorders - Drug Targets Manipulation of the Immune System for Cancer Defeat: A Focus on the T Cell Inhibitory Checkpoint Molecules
Current Medicinal Chemistry Cells and Mediators of Inflammation in Acute Pancreatitis
Clinical Anti-Inflammatory & Anti-Allergy Drugs (Discontinued) Vaccination Against High Blood Pressure
Current Pharmaceutical Design Recent Updates on the Association Between Alzheimer’s Disease and Vascular Dementia
Medicinal Chemistry Cardiovascular Magnetic Resonance for Evaluation of Heart Involvement in ANCA-Associated Vasculitis. A Luxury or a Valuable Diagnostic Tool?
Inflammation & Allergy - Drug Targets (Discontinued) Prophylactic Vaccine Approach for Colon and Pancreatic Cancers: Present and Future
Current Medicinal Chemistry Targeting the Mitochondrial Electron Transport Chain Complexes for the Induction of Apoptosis and Cancer Treatment
Current Pharmaceutical Biotechnology Mitochondria Sentencing About Cellular Life and Death: A Matter of Oxidative Stress
Current Pharmaceutical Design Molecular Chaperone Disorders: Defective Hsp60 in Neurodegeneration
Current Topics in Medicinal Chemistry Angiotensin-(1-7), Angiotensin-Converting Enzyme 2 and Mas Receptor in Rat Polycystic Ovaries
Protein & Peptide Letters Current Management of Intermittent Claudication: The Role of Pharmacological and Nonpharmacological Symptom-Directed Therapies
Current Vascular Pharmacology Endothelial Dysfunction and the Effects of TNF Inhibitors on the Endothelium in Psoriasis and Psoriatic Arthritis: A Systematic Review
Current Pharmaceutical Design Gene Therapy for Osteoinduction
Current Gene Therapy