期刊
NATURE STRUCTURAL & MOLECULAR BIOLOGY
卷 18, 期 1, 页码 85-87出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/nsmb.1958
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资金
- Association Francaise contre les Myopathies
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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