Published in Mol Ther on September 02, 2008
Synonymous but not the same: the causes and consequences of codon bias. Nat Rev Genet (2010) 5.37
Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice. Mol Ther (2010) 1.48
Therapeutic approaches to muscular dystrophy. Hum Mol Genet (2011) 1.30
Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy. Dis Model Mech (2015) 1.30
Synthetic biology: tools to design, build, and optimize cellular processes. J Biomed Biotechnol (2010) 1.26
Physiological characterization of muscle strength with variable levels of dystrophin restoration in mdx mice following local antisense therapy. Mol Ther (2010) 1.20
Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy. PLoS One (2012) 1.15
The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins. PLoS Genet (2010) 1.08
How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse. Hum Mol Genet (2015) 1.07
Gene therapy for retinal disease. Transl Res (2013) 1.03
Oversized AAV transductifon is mediated via a DNA-PKcs-independent, Rad51C-dependent repair pathway. Mol Ther (2013) 1.02
Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches. Nucleic Acids Res (2013) 0.91
Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy. Curr Pediatr Rep (2014) 0.89
Duchenne muscular dystrophy gene therapy in the canine model. Hum Gene Ther Clin Dev (2015) 0.86
Transcription factor rational design improves directed differentiation of human mesenchymal stem cells into skeletal myocytes. Mol Ther (2011) 0.85
The Rag2⁻Il2rb⁻Dmd⁻ mouse: a novel dystrophic and immunodeficient model to assess innovating therapeutic strategies for muscular dystrophies. Mol Ther (2013) 0.84
Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy. Pharmaceuticals (Basel) (2013) 0.82
Abnormal splicing switch of DMD's penultimate exon compromises muscle fibre maintenance in myotonic dystrophy. Nat Commun (2015) 0.81
Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors. Curr Gene Ther (2012) 0.81
Membrane-stabilizing copolymers confer marked protection to dystrophic skeletal muscle in vivo. Mol Ther Methods Clin Dev (2015) 0.81
The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer. Curr Gene Ther (2015) 0.80
Muscle fiber type-predominant promoter activity in lentiviral-mediated transgenic mouse. PLoS One (2011) 0.79
AAV Gene Therapy for Liver Disease. Hum Gene Ther (2016) 0.79
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy. Nat Commun (2017) 0.77
Full-length Dysferlin Transfer by the Hyperactive Sleeping Beauty Transposase Restores Dysferlin-deficient Muscle. Mol Ther Nucleic Acids (2016) 0.76
Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA. Mol Ther Methods Clin Dev (2015) 0.76
Conserved regions of the DMD 3' UTR regulate translation and mRNA abundance in cultured myotubes. Neuromuscul Disord (2014) 0.76
Dystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophies. Bosn J Basic Med Sci (2015) 0.76
Stable in Vivo Transgene Expression in Endothelial Cells with Helper-Dependent Adenovirus: Roles of Promoter and Interleukin-10. Hum Gene Ther (2016) 0.75
Glycogen storage disease type Ia mice with less than 2% of normal hepatic glucose-6-phosphatase-α activity restored are at risk of developing hepatic tumors. Mol Genet Metab (2017) 0.75
Restoration of dystrophin expression using the Sleeping Beauty transposon. PLoS Curr (2011) 0.75
Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice. Mol Ther Nucleic Acids (2016) 0.75
Nanotherapy for Duchenne muscular dystrophy. Wiley Interdiscip Rev Nanomed Nanobiotechnol (2017) 0.75
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
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther (2011) 2.06
Adenovirus vector vaccination induces expansion of memory CD4 T cells with a mucosal homing phenotype that are readily susceptible to HIV-1. Proc Natl Acad Sci U S A (2009) 1.95
Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice. Mol Ther (2010) 1.48
Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene. Biochem Biophys Res Commun (2002) 1.32
The neuroprotective effects of heat shock protein 27 overexpression in transgenic animals against kainate-induced seizures and hippocampal cell death. J Biol Chem (2003) 1.24
Poly(A) binding protein nuclear 1 levels affect alternative polyadenylation. Nucleic Acids Res (2012) 1.23
β-catenin as a potential key target for tumor suppression. Int J Cancer (2011) 1.23
Muscular dystrophies due to glycosylation defects: diagnosis and therapeutic strategies. Curr Opin Neurol (2011) 1.21
Relocalization of neuronal nitric oxide synthase (nNOS) as a marker for complete restoration of the dystrophin associated protein complex in skeletal muscle. Neuromuscul Disord (2003) 1.20
Physiological characterization of muscle strength with variable levels of dystrophin restoration in mdx mice following local antisense therapy. Mol Ther (2010) 1.20
Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice. Hum Gene Ther (2009) 1.19
Expression of opsin genes early in ocular development of humans and mice. Exp Eye Res (2003) 1.14
Characterization of the role of gamma2 R531G mutation in AMP-activated protein kinase in cardiac hypertrophy and Wolff-Parkinson-White syndrome. Am J Physiol Heart Circ Physiol (2005) 1.14
Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa micro-dystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre degeneration in nude/mdx mice. Hum Mol Genet (2002) 1.13
Zebrafish melanopsin: isolation, tissue localisation and phylogenetic position. Brain Res Mol Brain Res (2002) 1.12
Protective effects of heat shock protein 27 in a model of ALS occur in the early stages of disease progression. Neurobiol Dis (2007) 1.11
Molecular and phenotypic characterization of a mouse model of oculopharyngeal muscular dystrophy reveals severe muscular atrophy restricted to fast glycolytic fibres. Hum Mol Genet (2010) 1.10
Molecular, cellular and physiological investigation of myostatin propeptide-mediated muscle growth in adult mice. Neuromuscul Disord (2009) 1.09
Smooth muscle-specific dystrophin expression improves aberrant vasoregulation in mdx mice. Hum Mol Genet (2006) 1.09
Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases. Nucleic Acids Res (2012) 1.09
Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in. Hum Gene Ther (2013) 1.08
Extracellular release of the atheroprotective heat shock protein 27 is mediated by estrogen and competitively inhibits acLDL binding to scavenger receptor-A. Circ Res (2008) 1.08
In vivo fluorescence lifetime tomography of a FRET probe expressed in mouse. Biomed Opt Express (2011) 1.07
Heat shock protein 27 protects the heart against myocardial infarction. Basic Res Cardiol (2004) 1.06
Langerin negative dendritic cells promote potent CD8+ T-cell priming by skin delivery of live adenovirus vaccine microneedle arrays. Proc Natl Acad Sci U S A (2013) 1.05
Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice. Hum Gene Ther (2013) 1.04
Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment. Mol Ther (2010) 1.03
Hsp27 overexpression in the R6/2 mouse model of Huntington's disease: chronic neurodegeneration does not induce Hsp27 activation. Hum Mol Genet (2007) 1.03
Translational and regulatory challenges for exon skipping therapies. Hum Gene Ther (2014) 1.02
Heat shock protein 27 protects against atherogenesis via an estrogen-dependent mechanism: role of selective estrogen receptor beta modulation. Arterioscler Thromb Vasc Biol (2009) 1.02
New developments in the use of gene therapy to treat Duchenne muscular dystrophy. Expert Opin Biol Ther (2013) 1.01
Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy. Hum Gene Ther (2004) 1.01
Gene therapy for muscular dystrophy: current progress and future prospects. Expert Opin Biol Ther (2009) 1.01
Combination of myostatin pathway interference and dystrophin rescue enhances tetanic and specific force in dystrophic mdx mice. Mol Ther (2010) 1.01
RNAi-mediated knockdown of dystrophin expression in adult mice does not lead to overt muscular dystrophy pathology. Hum Mol Genet (2008) 0.99
Long-term systemic administration of unconjugated morpholino oligomers for therapeutic expression of dystrophin by exon skipping in skeletal muscle: implications for cardiac muscle integrity. Nucleic Acid Ther (2011) 0.98
Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene. Mol Ther (2009) 0.98
Metformin treatment has no beneficial effect in a dose-response survival study in the SOD1(G93A) mouse model of ALS and is harmful in female mice. PLoS One (2011) 0.98
Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog. J Gene Med (2011) 0.95
Atrophy, fibrosis, and increased PAX7-positive cells in pharyngeal muscles of oculopharyngeal muscular dystrophy patients. J Neuropathol Exp Neurol (2013) 0.95
Adeno-associated virus-8-mediated intravenous transfer of myostatin propeptide leads to systemic functional improvements of slow but not fast muscle. Rejuvenation Res (2009) 0.95
Transgenic overexpression of LARGE induces α-dystroglycan hyperglycosylation in skeletal and cardiac muscle. PLoS One (2010) 0.95
An IL-7 fusion protein that shows increased thymopoietic ability. J Immunol (2005) 0.94
Deregulation of the ubiquitin-proteasome system is the predominant molecular pathology in OPMD animal models and patients. Skelet Muscle (2011) 0.94
Microbubble stability is a major determinant of the efficiency of ultrasound and microbubble mediated in vivo gene transfer. Ultrasound Med Biol (2009) 0.93
Overexpression of heat shock protein 27 reduces cortical damage after cerebral ischemia. J Cereb Blood Flow Metab (2009) 0.92
Genetic therapeutic approaches for Duchenne muscular dystrophy. Hum Gene Ther (2012) 0.91
Current advances in cell therapy strategies for muscular dystrophies. Expert Opin Biol Ther (2011) 0.91
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
Structure and evolution of the teleost extraretinal rod-like opsin (errlo) and ocular rod opsin (rho) genes: is teleost rho a retrogene? J Exp Zool B Mol Dev Evol (2003) 0.88
The transgenic expression of LARGE exacerbates the muscle phenotype of dystroglycanopathy mice. Hum Mol Genet (2013) 0.88
Identification of a repressor in the first intron of the human alpha2(I) collagen gene (COL1A2). J Biol Chem (2005) 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
Long-term safety, tolerability, and clinical efficacy of quetiapine in adolescents: an open-label extension trial. J Child Adolesc Psychopharmacol (2003) 0.87
Role of a 5'-enhancer in the transcriptional regulation of the human endothelial cell protein C receptor gene. Blood (2006) 0.87
Preventing phosphorylation of dystroglycan ameliorates the dystrophic phenotype in mdx mouse. Hum Mol Genet (2012) 0.87
The human ClC-4 protein, a member of the CLC chloride channel/transporter family, is localized to the endoplasmic reticulum by its N-terminus. FASEB J (2006) 0.87
Cooperativity between far upstream enhancer and proximal promoter elements of the human {alpha}2(I) collagen (COL1A2) gene instructs tissue specificity in transgenic mice. J Biol Chem (2004) 0.86
Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials. Neuromuscul Disord (2010) 0.86
Electroporation for gene transfer to skeletal muscles: current status. BioDrugs (2004) 0.85
Transcriptomic analysis of dystrophin RNAi knockdown reveals a central role for dystrophin in muscle differentiation and contractile apparatus organization. BMC Genomics (2010) 0.85
Transcription factor rational design improves directed differentiation of human mesenchymal stem cells into skeletal myocytes. Mol Ther (2011) 0.85