Targeting nuclear RNA for in vivo correction of myotonic dystrophy (Q34459257)

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9 February 2020

title

Targeting nuclear RNA for in vivo correction of myotonic dystrophy (English)

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5

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author name string

Thurman M Wheeler

1

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9 February 2020

Andrew J Leger

2

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9 February 2020

Sanjay K Pandey

3

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9 February 2020

A Robert MacLeod

4

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9 February 2020

Seng H Cheng

6

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9 February 2020

Bruce M Wentworth

7

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C Frank Bennett

8

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Charles A Thornton

9

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9 February 2020

language of work or name

English

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publication date

1 August 2012

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volume

488

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issue

7409

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page(s)

111-115

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describes a project that uses

9 February 2020

ImageQuant

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11 June 2022

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inferred from PubMed Central ID database lookup

cites work

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Stabilization of expanded (CTG)•(CAG) repeats by antisense oligonucleotides

6 July 2018

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Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS

6 July 2018

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Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes

6 July 2018

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An upstream open reading frame and the context of the two AUG codons affect the abundance of mitochondrial and nuclear RNase H1

6 July 2018

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Aberrant alternative splicing and extracellular matrix gene expression in mouse models of myotonic dystrophy.

6 July 2018

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Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy.

6 July 2018

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Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA.

6 July 2018

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Transcriptional and post-transcriptional impact of toxic RNA in myotonic dystrophy

6 July 2018

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3' end processing of a long nuclear-retained noncoding RNA yields a tRNA-like cytoplasmic RNA

6 July 2018

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Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy

6 July 2018

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CTG trinucleotide repeat "big jumps": large expansions, small mice

6 July 2018

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Failure of MBNL1-dependent post-natal splicing transitions in myotonic dystrophy

6 July 2018

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Regulating gene expression through RNA nuclear retention

6 July 2018

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Translation of stable hepadnaviral mRNA cleavage fragments induced by the action of phosphorothioate-modified antisense oligodeoxynucleotides

6 July 2018

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Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs

6 July 2018

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A muscleblind knockout model for myotonic dystrophy

6 July 2018

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Expanded CUG repeats trigger aberrant splicing of ClC-1 chloride channel pre-mRNA and hyperexcitability of skeletal muscle in myotonic dystrophy

6 July 2018

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Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy

6 July 2018

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6 July 2018

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Transgenic mice carrying large human genomic sequences with expanded CTG repeat mimic closely the DM CTG repeat intergenerational and somatic instability.

6 July 2018

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Nucleocytoplasmic shuttling: a novel in vivo property of antisense phosphorothioate oligodeoxynucleotides.

6 July 2018

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A nonradioisotope biomedical assay for intact oligonucleotide and its chain-shortened metabolites used for determination of exposure and elimination half-life of antisense drugs in tissue

6 July 2018

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Expansion of a CUG trinucleotide repeat in the 3' untranslated region of myotonic dystrophy protein kinase transcripts results in nuclear retention of transcripts

6 July 2018

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Quantitation of phosphorothioate oligonucleotides in human plasma

6 July 2018

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Foci of trinucleotide repeat transcripts in nuclei of myotonic dystrophy cells and tissues

6 July 2018

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Rapid assembly and disassembly of complementary DNA strands through an equilibrium intermediate state mediated by A1 hnRNP protein

6 July 2018

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Molecular basis of myotonic dystrophy: expansion of a trinucleotide (CTG) repeat at the 3' end of a transcript encoding a protein kinase family member.

6 July 2018

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Expansion of an unstable DNA region and phenotypic variation in myotonic dystrophy

6 July 2018

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Pharmacokinetics of a tumor necrosis factor-alpha phosphorothioate 2'-O-(2-methoxyethyl) modified antisense oligonucleotide: comparison across species

28 July 2018

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Efficient reduction of target RNAs by small interfering RNA and RNase H-dependent antisense agents. A comparative analysis

21 January 2018

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Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

21 January 2018

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Solid phase synthesis of phosphorothioate oligonucleotides utilizing diethyldithiocarbonate disulfide (DDD) as an efficient sulfur transfer reagent

21 January 2018

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Mice transgenic for the human myotonic dystrophy region with expanded CTG repeats display muscular and brain abnormalities

21 January 2018

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What is principal component analysis?

21 January 2018

1 reference

CUG Repeats Present in Myotonin Kinase RNA Form Metastable “Slippery” Hairpins

21 January 2018

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12 December 2020

Myotonic dystrophy in transgenic mice expressing an expanded CUG repeat

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12 December 2020

Histological parameters for the quantitative assessment of muscular dystrophy in the mdx-mouse

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12 December 2020

Detection of an unstable fragment of DNA specific to individuals with myotonic dystrophy

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12 December 2020

Identifiers

DOI

10.1038/NATURE11362

1 reference

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Dimensions Publication ID

9 February 2020

0 references

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9 February 2020

PubMed publication ID

22859208

1 reference

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