Perspectives on gene therapy in myotonic dystrophy type 1. (Q37831858)

24 August 2017

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title

Perspectives on gene therapy in myotonic dystrophy type 1. (English)

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24 August 2017

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Jonathan J Magaña

1

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24 August 2017

Bulmaro Cisneros

series ordinal

2

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24 August 2017

publication date

16 December 2010

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Journal of Neuroscience Research

24 August 2017

published in

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24 August 2017

volume

89

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24 August 2017

issue

3

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24 August 2017

page(s)

275-285

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

24 August 2017

cites work

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Myotonic dystrophy is associated with a reduced level of RNA from the DMWD allele adjacent to the expanded repeat

21 January 2018

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A cell-based assay for aggregation inhibitors as therapeutics of polyglutamine-repeat disease and validation in Drosophila

21 January 2018

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A simple ligand that selectively targets CUG trinucleotide repeats and inhibits MBNL protein binding.

21 January 2018

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Therapeutic potential of splice-switching oligonucleotides

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RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform

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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.

21 January 2018

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Loss of the muscle-specific chloride channel in type 1 myotonic dystrophy due to misregulated alternative splicing

21 January 2018

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Correction of dystrophia myotonica type 1 pre-mRNA transcripts by artificial trans-splicing

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A small molecule microarray platform to select RNA internal loop-ligand interactions

21 January 2018

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Myotonic dystrophy: emerging mechanisms for DM1 and DM2.

21 January 2018

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MBNL1 is the primary determinant of focus formation and aberrant insulin receptor splicing in DM1

21 January 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

21 January 2018

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RNA pathogenesis of the myotonic dystrophies

21 January 2018

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MBNL1 and CUGBP1 modify expanded CUG-induced toxicity in a Drosophila model of myotonic dystrophy type 1.

21 January 2018

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Myotonic dystrophy 1 in the nervous system: from the clinic to molecular mechanisms

21 January 2018

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Intergenerational instability of the expanded CTG repeat in the DMPK gene: studies in human gametes and preimplantation embryos

21 January 2018

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Biologically active molecules that reduce polyglutamine aggregation and toxicity

21 January 2018

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Overexpression of MBNL1 fetal isoforms and modified splicing of Tau in the DM1 brain: two individual consequences of CUG trinucleotide repeats.

21 January 2018

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RNA leaching of transcription factors disrupts transcription in myotonic dystrophy

21 January 2018

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Three proteins, MBNL, MBLL and MBXL, co-localize in vivo with nuclear foci of expanded-repeat transcripts in DM1 and DM2 cells.

21 January 2018

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An unstable triplet repeat in a gene related to myotonic muscular dystrophy

21 January 2018

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Decreased expression of myotonin-protein kinase messenger RNA and protein in adult form of myotonic dystrophy.

21 January 2018

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Viral vector producing antisense RNA restores myotonic dystrophy myoblast functions

21 January 2018

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Genetic and chemical modifiers of a CUG toxicity model in Drosophila

21 January 2018

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Chemically induced increases and decreases in the rate of expansion of a CAG*CTG triplet repeat

21 January 2018

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Chemical modifiers of unstable expanded simple sequence repeats: what goes up, could come down

21 January 2018

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Selectable system for monitoring the instability of CTG/CAG triplet repeats in mammalian cells.

21 January 2018

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Comparison of different antisense strategies in mammalian cells using locked nucleic acids, 2'-O-methyl RNA, phosphorothioates and small interfering RNA.

21 January 2018

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Myotonic dystrophy protein kinase is expressed in embryonic myocytes and is required for myotube formation

21 January 2018

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99th ENMC international workshop: myotonic dystrophy: present management, future therapy. 9-11 November 2001, Naarden, The Netherlands.

21 January 2018

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Chemotherapeutically induced deletion of expanded triplet repeats

21 January 2018

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Genetic assays for measuring rates of (CAG).(CTG) repeat instability in Escherichia coli

21 January 2018

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Chemotherapeutic deletion of CTG repeats in lymphoblast cells from DM1 patients

21 January 2018

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Inhibition of huntingtin fibrillogenesis by specific antibodies and small molecules: implications for Huntington's disease therapy

21 January 2018

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Transgenic mice expressing CUG-BP1 reproduce splicing mis-regulation observed in myotonic dystrophy.

21 January 2018

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The roles of binding site arrangement and combinatorial targeting in microRNA repression of gene expression

21 January 2018

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Does (CUG)n repeat in DMPK mRNA 'paint' chromosome 19 to suppress distant genes to create the diverse phenotype of myotonic dystrophy?: A new hypothesis of long-range cis autosomal inactivation

21 January 2018

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Creatine-supplemented diet extends Purkinje cell survival in spinocerebellar ataxia type 1 transgenic mice but does not prevent the ataxic phenotype.

21 January 2018

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Myotonic dystrophy protein kinase (DMPK) and its role in the pathogenesis of myotonic dystrophy 1.

21 January 2018

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A muscleblind knockout model for myotonic dystrophy

21 January 2018

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Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophy

21 January 2018

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HnRNP H inhibits nuclear export of mRNA containing expanded CUG repeats and a distal branch point sequence

21 January 2018

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Oxidative stimuli affect polyglutamine aggregation and cell death in human mutant ataxin-1-expressing cells

21 January 2018

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Mice deficient in Six5 develop cataracts: implications for myotonic dystrophy

21 January 2018

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Heart-specific overexpression of CUGBP1 reproduces functional and molecular abnormalities of myotonic dystrophy type 1.

21 January 2018

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Antisense technologies. Improvement through novel chemical modifications

21 January 2018

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Increased steady-state levels of CUGBP1 in myotonic dystrophy 1 are due to PKC-mediated hyperphosphorylation

21 January 2018

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Hammerhead ribozyme-mediated destruction of nuclear foci in myotonic dystrophy myoblasts

21 January 2018

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Cytoplasmic and nuclear retained DMPK mRNAs are targets for RNA interference in myotonic dystrophy cells.

21 January 2018

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RNA-targeting approaches for neuromuscular diseases

21 January 2018

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

21 January 2018

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Myotonic dystrophy type 2 caused by a CCTG expansion in intron 1 of ZNF9

21 January 2018

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Molecular Effects of the CTG Repeats in Mutant Dystrophia Myotonica Protein Kinase Gene

21 January 2018

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Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles

21 January 2018

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Clinical and molecular aspects of the myotonic dystrophies: a review.

21 January 2018

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Myotonic dystrophy mutation: an unstable CTG repeat in the 3' untranslated region of the gene

21 January 2018

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21 January 2018

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Muscleblind localizes to nuclear foci of aberrant RNA in myotonic dystrophy types 1 and 2.

21 January 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

21 January 2018

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RNA repair using spliceosome-mediated RNA trans-splicing

21 January 2018

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Frequency and stability of the myotonic dystrophy type 1 premutation.

21 January 2018

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Woodchuck post-transcriptional element induces nuclear export of myotonic dystrophy 3' untranslated region transcripts

21 January 2018

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

21 January 2018

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Expandable DNA repeats and human disease.

21 January 2018

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

21 January 2018

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Molecular therapy in myotonic dystrophy: focus on RNA gain-of-function

21 January 2018

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Expanded CTG repeats within the DMPK 3' UTR causes severe skeletal muscle wasting in an inducible mouse model for myotonic dystrophy

21 January 2018

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

21 January 2018

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Slipped-strand DNAs formed by long (CAG)*(CTG) repeats: slipped-out repeats and slip-out junctions

21 January 2018

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Repeat instability: mechanisms of dynamic mutations

21 January 2018

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Disruption of splicing regulated by a CUG-binding protein in myotonic dystrophy

21 January 2018

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Ribozyme-mediated trans-splicing of a trinucleotide repeat.

21 January 2018

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Ribozymes as therapeutic tools for genetic disease.

21 January 2018

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A screen for drugs that protect against the cytotoxicity of polyglutamine-expanded androgen receptor.

21 January 2018

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Rational design of ligands targeting triplet repeating transcripts that cause RNA dominant disease: application to myotonic muscular dystrophy type 1 and spinocerebellar ataxia type 3.

21 January 2018

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RNA-mediated neuromuscular disorders

21 January 2018

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Specific and potent RNAi in the nucleus of human cells

21 January 2018

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Heterozygous loss of Six5 in mice is sufficient to cause ocular cataracts

21 January 2018

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Insulin receptor splicing alteration in myotonic dystrophy type 2.

21 January 2018

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Systemically delivered antisense oligomers upregulate gene expression in mouse tissues

21 January 2018

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Myotonic dystrophies type 1 and 2: a summary on current aspects.

21 January 2018

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Dysregulation of human brain microtubule-associated tau mRNA maturation in myotonic dystrophy type 1.

21 January 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.

21 January 2018

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Inhibition of polyglutamine aggregation in R6/2 HD brain slices-complex dose-response profiles

21 January 2018

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Defining early steps in mRNA transport: mutant mRNA in myotonic dystrophy type I is blocked at entry into SC-35 domains

21 January 2018

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Histone deacetylase inhibitors arrest polyglutamine-dependent neurodegeneration in Drosophila

21 January 2018

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A novel therapeutic strategy for polyglutamine diseases by stabilizing aggregation-prone proteins with small molecules

21 January 2018

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

21 January 2018

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AAV-directed muscular dystrophy gene therapy

21 January 2018

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Expanded CUG repeat RNAs form hairpins that activate the double-stranded RNA-dependent protein kinase PKR

21 January 2018

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Novel proteins with binding specificity for DNA CTG repeats and RNA CUG repeats: implications for myotonic dystrophy

21 January 2018

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RNA CUG repeats sequester CUGBP1 and alter protein levels and activity of CUGBP1

21 January 2018

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Elevation of RNA-binding protein CUGBP1 is an early event in an inducible heart-specific mouse model of myotonic dystrophy

21 January 2018

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PKC inhibition ameliorates the cardiac phenotype in a mouse model of myotonic dystrophy type 1.

21 January 2018

1 reference

Reversal of a full-length mutant huntingtin neuronal cell phenotype by chemical inhibitors of polyglutamine-mediated aggregation

21 January 2018

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Role of RNA structure in regulating pre-mRNA splicing

21 January 2018

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Pentamidine reverses the splicing defects associated with myotonic dystrophy.

21 January 2018

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Inhibiting caspase cleavage of huntingtin reduces toxicity and aggregate formation in neuronal and nonneuronal cells

21 January 2018

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Myotonic dystrophy: therapeutic strategies for the future

21 January 2018

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

21 January 2018

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Characterization of the cDNA and the gene encoding murine adenylosuccinate lyase

21 January 2018

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Modulating the expression of disease genes with RNA-based therapy.

21 January 2018

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RNA-targeted splice-correction therapy for neuromuscular disease

21 January 2018

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Cell-penetrating peptides as transporters for morpholino oligomers: effects of amino acid composition on intracellular delivery and cytotoxicity

21 January 2018

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A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.

21 January 2018

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Potentiation of NMDA receptor-mediated excitotoxicity linked with intrinsic apoptotic pathway in YAC transgenic mouse model of Huntington's disease

21 January 2018

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A potent small molecule inhibits polyglutamine aggregation in Huntington's disease neurons and suppresses neurodegeneration in vivo

21 January 2018

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21 January 2018

Identifiers

DOI

10.1002/JNR.22551

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24 August 2017

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