Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard
Nantes
France

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MicroRNAs in Genetic Disease: Rethinking the Dosage

Author(s): Alexandra Henrion-Caude, Muriel Girard, Jeanne Amiel.

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.

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Article Details

VOLUME: 12
ISSUE: 4
Year: 2012
Page: [292 - 300]
Pages: 9
DOI: 10.2174/156652312802083602
Price: $58