Rna therapy for myotonic dystrophy


Rna therapy for myotonic dystrophy

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Access through your institution Buy or subscribe Working in a mouse model of myotonic dystrophy type 1 (DM1), the authors designed antisense oligonucleotides (ASOs) that target nuclear


retained transcripts of a human _ACTAC1_ transgene containing an expanded CUG tract (CUGexp). The ASOs were optimized for processing by the RNAaseH pathway. Injecting these mice with ASOs


caused a rapid knockdown of CUGexp as well as changes in the transcriptome that were consistent with the splice factor muscleblind being released from CUGexp sequestration, which is a


mechanism by which the aberrant transcripts are thought to cause disease. Histological features of the disease were also corrected, and this is a promising advance for therapy. This is a


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during checkout ADDITIONAL ACCESS OPTIONS: * Log in * Learn about institutional subscriptions * Read our FAQs * Contact customer support ORIGINAL RESEARCH PAPER * Wheeler, T. M. et al.


Targeting nuclear RNA for _in vivo_ correction of myotonic dystrophy. _Nature_ 488, 111–115 (2012) Article  CAS  PubMed  PubMed Central  Google Scholar  Download references Authors * Hannah


Stower View author publications You can also search for this author inPubMed Google Scholar RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Stower, H.


RNA therapy for myotonic dystrophy. _Nat Rev Genet_ 13, 598 (2012). https://doi.org/10.1038/nrg3323 Download citation * Published: 14 August 2012 * Issue Date: September 2012 * DOI:


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