Published in Hum Mol Genet on April 01, 2002
Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med (2010) 4.28
Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. Mol Ther (2005) 2.08
Treatment of human disease by adeno-associated viral gene transfer. Hum Genet (2006) 1.84
Viral serotype and the transgene sequence influence overlapping adeno-associated viral (AAV) vector-mediated gene transfer in skeletal muscle. J Gene Med (2006) 1.36
Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice. Mol Ther (2008) 1.29
Viral-mediated gene therapy for the muscular dystrophies: successes, limitations and recent advances. Biochim Biophys Acta (2006) 1.25
AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy. Mol Ther (2013) 1.03
Approaching a new age in Duchenne muscular dystrophy treatment. Neurotherapeutics (2008) 0.88
Duchenne muscular dystrophy gene therapy in the canine model. Hum Gene Ther Clin Dev (2015) 0.86
Development of viral vectors for use in cardiovascular gene therapy. Viruses (2010) 0.81
Multi-parametric MRI at 14T for muscular dystrophy mice treated with AAV vector-mediated gene therapy. PLoS One (2015) 0.81
Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice. Mol Ther Nucleic Acids (2015) 0.80
Phosphorylation within the cysteine-rich region of dystrophin enhances its association with β-dystroglycan and identifies a potential novel therapeutic target for skeletal muscle wasting. Hum Mol Genet (2014) 0.77
Conserved regions of the DMD 3' UTR regulate translation and mRNA abundance in cultured myotubes. Neuromuscul Disord (2014) 0.76
Genome Editing Gene Therapy for Duchenne Muscular Dystrophy. J Neuromuscul Dis (2015) 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
Codon and mRNA sequence optimization of microdystrophin transgenes improves expression and physiological outcome in dystrophic mdx mice following AAV2/8 gene transfer. Mol Ther (2008) 1.24
Poly(A) binding protein nuclear 1 levels affect alternative polyadenylation. Nucleic Acids Res (2012) 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
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
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
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
Deregulation of the ubiquitin-proteasome system is the predominant molecular pathology in OPMD animal models and patients. Skelet Muscle (2011) 0.94
An IL-7 fusion protein that shows increased thymopoietic ability. J Immunol (2005) 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
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
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
Heat shock protein 27 overexpression mitigates cytokine-induced islet apoptosis and streptozotocin-induced diabetes. Endocrinology (2009) 0.85
Long-term episomal transgene expression from mitotically stable integration-deficient lentiviral vectors. Hum Gene Ther (2014) 0.84
Production of non viral DNA vectors. Curr Gene Ther (2010) 0.84
Stable micro-dystrophin gene transfer using an integrating adeno-retroviral hybrid vector ameliorates the dystrophic pathology in mdx mouse muscle. Hum Mol Genet (2002) 0.83
Identification of the key regions within the mouse pro-alpha 2(I) collagen gene far-upstream enhancer. J Biol Chem (2001) 0.83
In silico characterisation and chromosomal localisation of human RRH (peropsin)--implications for opsin evolution. BMC Genomics (2003) 0.83
Human apolipoprotein E expression from mouse skeletal muscle by electrotransfer of nonviral DNA (plasmid) and pseudotyped recombinant adeno-associated virus (AAV2/7). Hum Gene Ther (2008) 0.82
Langerhans cells are more efficiently transduced than dermal dendritic cells by adenovirus vectors expressing either group C or group B fibre protein: implications for mucosal vaccines. Eur J Immunol (2005) 0.82
Towards a therapeutic inhibition of dystrophin exon 23 splicing in mdx mouse muscle induced by antisense oligoribonucleotides (splicomers): target sequence optimisation using oligonucleotide arrays. J Gene Med (2004) 0.82
APOE epsilon3 gene transfer attenuates brain damage after experimental stroke. J Cereb Blood Flow Metab (2006) 0.82
Fragmentation of SIV-gag vaccine induces broader T cell responses. PLoS One (2012) 0.81
Codon optimization of the microdystrophin gene for Duchene muscular dystrophy gene therapy. Methods Mol Biol (2011) 0.81
Effect of albumin and dextrose concentration on ultrasound and microbubble mediated gene transfection in vivo. Ultrasound Med Biol (2012) 0.81
Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD). Curr Gene Ther (2015) 0.80
A new extensively characterised conditionally immortal muscle cell-line for investigating therapeutic strategies in muscular dystrophies. PLoS One (2011) 0.80
Failure to generate atheroprotective apolipoprotein AI phenotypes using synthetic RNA/DNA oligonucleotides (chimeraplasts). J Gene Med (2003) 0.80
Highly potent delivery method of gp160 envelope vaccine combining lentivirus-like particles and DNA electrotransfer. J Control Release (2012) 0.79
Apolipoprotein E delivery by peritoneal implantation of encapsulated recombinant cells improves the hyperlipidaemic profile in apoE-deficient mice. Biochim Biophys Acta (2005) 0.79
Influence of needle gauge on in vivo ultrasound and microbubble-mediated gene transfection. Ultrasound Med Biol (2011) 0.79
Preliminary evaluation of a self-complementary AAV2/8 vector for hepatic gene transfer of human apoE3 to inhibit atherosclerotic lesion development in apoE-deficient mice. Atherosclerosis (2008) 0.78
Novel cationic carotenoid lipids as delivery vectors of antisense oligonucleotides for exon skipping in Duchenne muscular dystrophy. Molecules (2012) 0.78
Optimizing antisense oligonucleotides using phosphorodiamidate morpholino oligomers. Methods Mol Biol (2012) 0.78
Bioinformatic and functional optimization of antisense phosphorodiamidate morpholino oligomers (PMOs) for therapeutic modulation of RNA splicing in muscle. Methods Mol Biol (2011) 0.78
Gene repair and mutagenesis mediated by chimeric RNA-DNA oligonucleotides: chimeraplasty for gene therapy and conversion of single nucleotide polymorphisms (SNPs). Biochim Biophys Acta (2002) 0.78
Nuclear entrapment and extracellular depletion of PCOLCE is associated with muscle degeneration in oculopharyngeal muscular dystrophy. BMC Neurol (2013) 0.78
Gene delivery to dystrophic muscle. Methods Mol Biol (2008) 0.78
Hox proteins drive cell segregation and non-autonomous apical remodelling during hindbrain segmentation. Development (2014) 0.76
Förster resonance energy transfer imaging in vivo with approximated radiative transfer equation. Appl Opt (2011) 0.75
Adenovirus-based targeting in myoblasts is hampered by nonhomologous vector integration. Hum Gene Ther (2008) 0.75
Restoration of dystrophin expression using the Sleeping Beauty transposon. PLoS Curr (2011) 0.75
Adeno-associated virus serotypes 7 and 8 outperform serotype 9 in expressing atheroprotective human apoE3 from mouse skeletal muscle. Metabolism (2010) 0.75
Sustained transgene expression using non-viral enzymatic systems for stable chromosomal integration. Curr Gene Ther (2008) 0.75
Getting to the heart of things in the field of cardiovascular gene therapy. Hum Gene Ther (2011) 0.75
Activity of different vaccine-associated promoter elements in human dendritic cells. Immunol Lett (2007) 0.75
The future of Duchenne muscular dystrophy gene therapy: shrinking the dystrophin gene. Curr Opin Mol Ther (2002) 0.75