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.
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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 |
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