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July 16, 2009ScienceOpen Access

Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA

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Population

Transgenic mouse model of myotonic dystrophy

Design

Preclinical

Authors

TWThurman M. WheelerHarvard UniversityKSKrzysztof SobczakGene Therapy LaboratoryJLJohn D. LueckUniversity of Rochester Medical Center

Discussion

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Implication

CAG25 reverses molecular defects in a myotonic dystrophy mouse model; leaves open whether antisense oligonucleotides will prove safe or effective in patients.

Structured PICO

P
Population
Transgenic mouse model of myotonic dystrophy
I
Intervention
Morpholino antisense oligonucleotide, CAG25
O
Outcome
Reversal of derangements of myotonic dystrophy, including dispersion of nuclear foci of CUGexp RNA, reduction of toxic RNA burden, correction of alternative splicing regulation, and restoration of ion channel functionsurrogate

Antisense oligonucleotides can reverse the molecular and functional derangements of myotonic dystrophy in a transgenic mouse model by blocking pathogenic RNA-protein interactions.

Cite This Study

Wheeler et al. (2009) studied this question.

synapsesocial.com/papers/6a710276f44fa9f079dec639https://doi.org/10.1126/science.1173110
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophy2006 · 377 citations
  2. 2Expansion of a CUG trinucleotide repeat in the 3′ untranslated region of myotonic dystrophy protein kinase transcripts results in nuclear retention of transcripts1997 · 476 citations
  3. 3Identification of a (CUG)n Triplet Repeat RNA-Binding Protein and Its Expression in Myotonic Dystrophy1996 · 469 citations
  4. 4Myotonic Dystrophy Type 2 Caused by a CCTG Expansion in Intron 1 of ZNF92001 · 1,242 citations
  5. 5CUG Repeats Present in Myotonin Kinase RNA Form Metastable “Slippery” Hairpins1997 · 178 citations