Published in J Gene Med on March 01, 2009
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol (2009) 5.35
RNA therapeutics: beyond RNA interference and antisense oligonucleotides. Nat Rev Drug Discov (2012) 3.70
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther (2011) 2.06
Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers. Nat Med (2015) 2.04
Targeting RNA to treat neuromuscular disease. Nat Rev Drug Discov (2011) 1.89
Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches. Nat Rev Genet (2013) 1.70
The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy. Mol Ther (2010) 1.38
Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model. Mol Ther (2010) 1.32
Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy. Mol Ther (2011) 1.30
Emerging strategies for cell and gene therapy of the muscular dystrophies. Expert Rev Mol Med (2009) 1.29
Genotype and phenotype characterization in a large dystrophinopathic cohort with extended follow-up. J Neurol (2011) 1.28
Physiological characterization of muscle strength with variable levels of dystrophin restoration in mdx mice following local antisense therapy. Mol Ther (2010) 1.20
Progress in therapeutic antisense applications for neuromuscular disorders. Eur J Hum Genet (2009) 1.19
The effects of low levels of dystrophin on mouse muscle function and pathology. PLoS One (2012) 1.16
Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO. Mol Ther (2010) 1.10
Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping. Hum Mol Genet (2012) 1.10
Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice. EMBO Mol Med (2013) 1.01
A chemical view of oligonucleotides for exon skipping and related drug applications. Nucleic Acid Ther (2013) 1.00
Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse. Mol Ther (2010) 0.98
Persistent dystrophin protein restoration 90 days after a course of intraperitoneally administered naked 2'OMePS AON and ZM2 NP-AON complexes in mdx mice. J Biomed Biotechnol (2012) 0.97
Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice. Sci Rep (2015) 0.94
Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy. EMBO Mol Med (2015) 0.93
Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy. BMC Med Genomics (2011) 0.92
Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids (2012) 0.89
Development of therapeutic splice-switching oligonucleotides. Hum Gene Ther (2014) 0.88
Antisense oligonucleotide-mediated MDM4 exon 6 skipping impairs tumor growth. J Clin Invest (2015) 0.87
Antisense therapy in neurology. J Pers Med (2013) 0.87
A prospective study in the rational design of efficient antisense oligonucleotides for exon skipping in the DMD gene. Hum Gene Ther (2012) 0.86
Effective exon skipping and dystrophin restoration by 2'-o-methoxyethyl antisense oligonucleotide in dystrophin-deficient mice. PLoS One (2013) 0.86
Biodistribution and molecular studies on orally administered nanoparticle-AON complexes encapsulated with alginate aiming at inducing dystrophin rescue in mdx mice. Biomed Res Int (2013) 0.85
Antisense-mediated RNA targeting: versatile and expedient genetic manipulation in the brain. Front Mol Neurosci (2011) 0.84
Splice-switching antisense oligonucleotides as therapeutic drugs. Nucleic Acids Res (2016) 0.84
Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases. Nucleic Acid Ther (2014) 0.83
Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models. Mol Ther Nucleic Acids (2012) 0.83
Identification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screening. PLoS One (2009) 0.83
Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy. Skelet Muscle (2011) 0.82
Development and Application of an Ultrasensitive Hybridization-Based ELISA Method for the Determination of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides. Nucleic Acid Ther (2015) 0.81
Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials. Am J Transl Res (2016) 0.80
Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching. BMC Med Genet (2011) 0.80
MYBPC3 in hypertrophic cardiomyopathy: from mutation identification to RNA-based correction. Pflugers Arch (2013) 0.80
Antisense mediated exon skipping therapy for duchenne muscular dystrophy (DMD). Artif DNA PNA XNA (2011) 0.78
Towards Fluorescence In Vivo Hybridization (FIVH) Detection of H. pylori in Gastric Mucosa Using Advanced LNA Probes. PLoS One (2015) 0.78
Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides. Mol Ther Nucleic Acids (2014) 0.77
Preclinical studies on intestinal administration of antisense oligonucleotides as a model for oral delivery for treatment of duchenne muscular dystrophy. Mol Ther Nucleic Acids (2014) 0.77
Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in Duchenne muscular dystrophy. PLoS One (2014) 0.77
Prednisolone treatment does not interfere with 2'-O-methyl phosphorothioate antisense-mediated exon skipping in Duchenne muscular dystrophy. Hum Gene Ther (2012) 0.77
Oligonucleotide-induced alternative splicing of serotonin 2C receptor reduces food intake. EMBO Mol Med (2016) 0.77
Genome Editing Gene Therapy for Duchenne Muscular Dystrophy. J Neuromuscul Dis (2015) 0.75
Electroporation Enhanced Effect of Dystrophin Splice Switching PNA Oligomers in Normal and Dystrophic Muscle. Mol Ther Nucleic Acids (2015) 0.75
Circulating Biomarkers for Duchenne Muscular Dystrophy. J Neuromuscul Dis (2015) 0.75
The effect of 6-thioguanine on alternative splicing and antisense-mediated exon skipping treatment for duchenne muscular dystrophy. PLoS Curr (2012) 0.75
Antisense oligonucleotide induction of progerin in human myogenic cells. PLoS One (2014) 0.75
Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle. Nat Commun (2017) 0.75
Deep sequencing-based expression analysis shows major advances in robustness, resolution and inter-lab portability over five microarray platforms. Nucleic Acids Res (2008) 10.10
Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med (2007) 7.29
Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med (2011) 5.91
Phenotypically concordant and discordant monozygotic twins display different DNA copy-number-variation profiles. Am J Hum Genet (2008) 5.53
Hypomethylation of D4Z4 in 4q-linked and non-4q-linked facioscapulohumeral muscular dystrophy. Nat Genet (2003) 3.60
Mutations in SWI/SNF chromatin remodeling complex gene ARID1B cause Coffin-Siris syndrome. Nat Genet (2012) 2.63
Reproducibility of high-throughput mRNA and small RNA sequencing across laboratories. Nat Biotechnol (2013) 2.53
Facioscapulohumeral muscular dystrophy is uniquely associated with one of the two variants of the 4q subtelomere. Nat Genet (2002) 2.42
Genetic heterogeneity in Rubinstein-Taybi syndrome: mutations in both the CBP and EP300 genes cause disease. Am J Hum Genet (2005) 2.40
Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy. Proc Natl Acad Sci U S A (2009) 2.32
Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum Mol Genet (2003) 2.17
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther (2011) 2.06
Deep sequencing to reveal new variants in pooled DNA samples. Hum Mutat (2009) 2.04
Advances in Duchenne muscular dystrophy gene therapy. Nat Rev Genet (2003) 1.96
Copy number variation in regions flanked (or unflanked) by duplicons among patients with developmental delay and/or congenital malformations; detection of reciprocal and partial Williams-Beuren duplications. Eur J Hum Genet (2006) 1.93
Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications. RNA (2007) 1.78
An immune response network associated with blood lipid levels. PLoS Genet (2010) 1.74
Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy. Neuromuscul Disord (2002) 1.72
Frequency of new copy number variation in humans. Nat Genet (2005) 1.71
Comprehensive detection of genomic duplications and deletions in the DMD gene, by use of multiplex amplifiable probe hybridization. Am J Hum Genet (2002) 1.55
Small N-terminal mutant huntingtin fragments, but not wild type, are mainly present in monomeric form: Implications for pathogenesis. Exp Neurol (2005) 1.53
Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense. Am J Hum Genet (2003) 1.52
Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms. Mol Ther (2008) 1.48
Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides. Mol Ther (2004) 1.46
Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons. Mol Ther (2006) 1.38
Phage display screening without repetitious selection rounds. Anal Biochem (2011) 1.36
Differential requirements for VirB1 and VirB2 during Brucella abortus infection. Infect Immun (2004) 1.33
Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model. Mol Ther (2010) 1.32
CORE_TF: a user-friendly interface to identify evolutionary conserved transcription factor binding sites in sets of co-regulated genes. BMC Bioinformatics (2008) 1.31
The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations. Hum Mutat (2015) 1.26
Splice modulating therapies for human disease. Cell (2012) 1.26
Human papillomavirus deregulates the response of a cellular network comprising of chemotactic and proinflammatory genes. PLoS One (2011) 1.26
Integrated analysis of DNA copy number and gene expression microarray data using gene sets. BMC Bioinformatics (2009) 1.25
Genome-wide assessment of differential roles for p300 and CBP in transcription regulation. Nucleic Acids Res (2010) 1.25
Prevention of oculopharyngeal muscular dystrophy-associated aggregation of nuclear polyA-binding protein with a single-domain intracellular antibody. Hum Mol Genet (2005) 1.19
Progress in therapeutic antisense applications for neuromuscular disorders. Eur J Hum Genet (2009) 1.19
Longitudinal evaluation of "presymptomatic" carriers of Huntington's disease. J Neuropsychiatry Clin Neurosci (2007) 1.18
Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials. Brain (2011) 1.17
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Mol Ther (2013) 1.16
The effects of low levels of dystrophin on mouse muscle function and pathology. PLoS One (2012) 1.16
Human papillomavirus (HPV) upregulates the cellular deubiquitinase UCHL1 to suppress the keratinocyte's innate immune response. PLoS Pathog (2013) 1.15
The therapeutic potential of antisense-mediated exon skipping. Curr Opin Mol Ther (2008) 1.15
Novel protein-protein interactions inferred from literature context. PLoS One (2009) 1.13
Tissue-specific transcript annotation and expression profiling with complementary next-generation sequencing technologies. Nucleic Acids Res (2010) 1.13
Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice. FASEB J (2013) 1.11
The Pathogenesis and Therapy of Muscular Dystrophies. Annu Rev Genomics Hum Genet (2015) 1.08
Diagnosis of genetic abnormalities in developmentally delayed patients: a new strategy combining MLPA and array-CGH. Am J Med Genet A (2007) 1.08
Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy. BMC Med Genet (2007) 1.08
Intensity-based analysis of two-colour microarrays enables efficient and flexible hybridization designs. Nucleic Acids Res (2004) 1.08
Targeting several CAG expansion diseases by a single antisense oligonucleotide. PLoS One (2011) 1.08
Generation and characterization of transgenic mice with the full-length human DMD gene. J Biol Chem (2007) 1.07
Accurate quantification of dystrophin mRNA and exon skipping levels in duchenne muscular dystrophy. Lab Invest (2010) 1.01
Serum protein profiling in mice: identification of Factor XIIIa as a potential biomarker for muscular dystrophy. Proteomics (2008) 1.00
Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy. Mol Ther (2009) 1.00
Exonic sequences provide better targets for antisense oligonucleotides than splice site sequences in the modulation of Duchenne muscular dystrophy splicing. Oligonucleotides (2010) 1.00
Comparison of skeletal muscle pathology and motor function of dystrophin and utrophin deficient mouse strains. Neuromuscul Disord (2012) 0.99
Large-scale gene expression analysis of human skeletal myoblast differentiation. Neuromuscul Disord (2004) 0.98
Cognitive and motor functioning in gene carriers for Huntington's disease: a baseline study. J Neuropsychiatry Clin Neurosci (2003) 0.97
Strong aggregation and increased toxicity of polyleucine over polyglutamine stretches in mammalian cells. Hum Mol Genet (2002) 0.97
Fibronectin is a serum biomarker for Duchenne muscular dystrophy. Proteomics Clin Appl (2014) 0.96
Exon skipping and gene transfer restore dystrophin expression in human induced pluripotent stem cells-cardiomyocytes harboring DMD mutations. Stem Cells Dev (2013) 0.95
Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy. BMC Med Genet (2008) 0.95
Deregulation of the ubiquitin-proteasome system is the predominant molecular pathology in OPMD animal models and patients. Skelet Muscle (2011) 0.94
A 3 months mild functional test regime does not affect disease parameters in young mdx mice. Neuromuscul Disord (2010) 0.94
Embryonic expression patterns of the Drosophila dystrophin-associated glycoprotein complex orthologs. Gene Expr Patterns (2004) 0.93
Mutant huntingtin represses CBP, but not p300, by binding and protein degradation. Mol Cell Neurosci (2005) 0.93
Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle. Hum Mol Genet (2013) 0.93
Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy. BMC Med Genomics (2011) 0.92
Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translational. PLoS Curr (2013) 0.92