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

  title={Selective silencing of mutated mRNAs in DM1 by using modified hU7-snRNAs},
  author={Virginie François and Arnaud F. Klein and Cyriaque Beley and Arnaud Jollet and Camille Lemercier and Luis Gonz{\'a}lez Garc{\'i}a and Denis Furling},
  journal={Nature Structural &Molecular Biology},
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… CONTINUE READING


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