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. 2011 Jan;18(1):85-7.
doi: 10.1038/nsmb.1958. Epub 2010 Dec 26.

Selective silencing of mutated mRNAs in DM1 by using modified hU7-snRNAs

Virginie François  1 , Arnaud F Klein, Cyriaque Beley, Arnaud Jollet, Camille Lemercier, Luis Garcia, Denis Furling
Affiliations

Selective silencing of mutated mRNAs in DM1 by using modified hU7-snRNAs

Virginie François et al. Nat Struct Mol Biol. 2011 Jan.

Abstract

We describe a function for modified human U7 small nuclear RNAs (hU7-snRNAs) distinct from modification of pre-mRNA splicing events. Engineered hU7-snRNAs containing a poly-CAG antisense sequence targeting the expanded CUG repeats of mutant DMPK transcripts in myotonic dystrophy caused specific degradation of pathogenic DMPK mRNAs without affecting the products of wild-type DMPK alleles. Abolition of the RNA gain-of-function toxicity that is responsible for pathogenesis supports the use of hU7-snRNAs for gene silencing in RNA-dominant disorders in which expanded repeats are expressed.

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