Published in Mol Ther Nucleic Acids on May 17, 2017
Linear models and empirical bayes methods for assessing differential expression in microarray experiments. Stat Appl Genet Mol Biol (2004) 101.91
Exploration, normalization, and summaries of high density oligonucleotide array probe level data. Biostatistics (2003) 100.88
Controlling the false discovery rate in behavior genetics research. Behav Brain Res (2001) 21.82
Myotonic dystrophy in transgenic mice expressing an expanded CUG repeat. Science (2000) 5.85
Foci of trinucleotide repeat transcripts in nuclei of myotonic dystrophy cells and tissues. J Cell Biol (1995) 4.04
Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA. Science (2009) 3.62
A postnatal switch of CELF and MBNL proteins reprograms alternative splicing in the developing heart. Proc Natl Acad Sci U S A (2008) 3.33
Increased steady-state levels of CUGBP1 in myotonic dystrophy 1 are due to PKC-mediated hyperphosphorylation. Mol Cell (2007) 3.26
affylmGUI: a graphical user interface for linear modeling of single channel microarray data. Bioinformatics (2006) 2.74
RNA CUG repeats sequester CUGBP1 and alter protein levels and activity of CUGBP1. J Biol Chem (2000) 2.65
Targeting nuclear RNA for in vivo correction of myotonic dystrophy. Nature (2012) 2.63
Transgenic mice expressing CUG-BP1 reproduce splicing mis-regulation observed in myotonic dystrophy. Hum Mol Genet (2005) 2.59
Mice lacking the myotonic dystrophy protein kinase develop a late onset progressive myopathy. Nat Genet (1996) 2.46
Mice transgenic for the human myotonic dystrophy region with expanded CTG repeats display muscular and brain abnormalities. Hum Mol Genet (2001) 2.08
Interaction of muscleblind, CUG-BP1 and hnRNP H proteins in DM1-associated aberrant IR splicing. EMBO J (2006) 1.80
Cytoplasmic and nuclear retained DMPK mRNAs are targets for RNA interference in myotonic dystrophy cells. J Biol Chem (2005) 1.64
Short antisense oligonucleotides with novel 2'-4' conformationaly restricted nucleoside analogues show improved potency without increased toxicity in animals. J Med Chem (2009) 1.61
Nuclear RNA foci in the heart in myotonic dystrophy. Circ Res (2005) 1.57
Pharmacokinetics of a tumor necrosis factor-alpha phosphorothioate 2'-O-(2-methoxyethyl) modified antisense oligonucleotide: comparison across species. Drug Metab Dispos (2003) 1.57
Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice. Mol Ther (2011) 1.32
Peripheral androgen receptor gene suppression rescues disease in mouse models of spinal and bulbar muscular atrophy. Cell Rep (2014) 1.32
Defective satellite cells in congenital myotonic dystrophy. Hum Mol Genet (2001) 1.29
Misregulation of alternative splicing causes pathogenesis in myotonic dystrophy. Prog Mol Subcell Biol (2006) 1.16
Clinical pharmacological properties of mipomersen (Kynamro), a second generation antisense inhibitor of apolipoprotein B. Br J Clin Pharmacol (2013) 1.16
Defective mRNA in myotonic dystrophy accumulates at the periphery of nuclear splicing speckles. Genes Cells (2007) 1.16
Maturational arrest of fetal muscle in neonatal myotonic dystrophy. A pathologic study of four cases. Arch Neurol (1976) 1.14
Myotonic dystrophy. Neurol Clin (2014) 1.08
Molecular, physiological, and motor performance defects in DMSXL mice carrying >1,000 CTG repeats from the human DM1 locus. PLoS Genet (2012) 1.01
Immaturity of muscle fibers in the congenital form of myotonic dystrophy: its consequences and its origin. J Neurol Sci (1988) 0.98
Preclinical evaluation of the toxicological effects of a novel constrained ethyl modified antisense compound targeting signal transducer and activator of transcription 3 in mice and cynomolgus monkeys. Nucleic Acid Ther (2013) 0.95
Antisense oligonucleotide treatment ameliorates alpha-1 antitrypsin-related liver disease in mice. J Clin Invest (2013) 0.94
Spinal muscular atrophy approval boosts antisense drugs. Nat Biotechnol (2017) 0.92
Effects of ageing on enzyme-histochemical, morphometrical and contractile properties of the soleus muscle in the rat. J Neurol Sci (1989) 0.91
Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts. Nucleic Acids Res (2015) 0.88
Progressive skeletal muscle weakness in transgenic mice expressing CTG expansions is associated with the activation of the ubiquitin-proteasome pathway. Neuromuscul Disord (2010) 0.87
Type 2 myotonic dystrophy can be predicted by the combination of type 2 muscle fiber central nucleation and scattered atrophy. J Neuropathol Exp Neurol (2008) 0.86
RBFOX1 cooperates with MBNL1 to control splicing in muscle, including events altered in myotonic dystrophy type 1. PLoS One (2014) 0.85
Myotonic dystrophy CTG expansion affects synaptic vesicle proteins, neurotransmission and mouse behaviour. Brain (2013) 0.84
Identification and Characterization of Modified Antisense Oligonucleotides Targeting DMPK in Mice and Nonhuman Primates for the Treatment of Myotonic Dystrophy Type 1. J Pharmacol Exp Ther (2015) 0.82
Peripheral neuropathy is linked to a severe form of myotonic dystrophy in transgenic mice. J Neuropathol Exp Neurol (2011) 0.82
Effects on murine behavior and lifespan of selectively decreasing expression of mutant huntingtin allele by supt4h knockdown. PLoS Genet (2015) 0.80
Cell membrane integrity in myotonic dystrophy type 1: implications for therapy. PLoS One (2015) 0.79