Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents.
暂无分享,去创建一个
[1] Kevin M Flanigan,et al. The Muscular Dystrophies , 1999, Seminars in Neurology.
[2] B. Nico,et al. Evaluation of potential synergistic action of a combined treatment with alpha‐methyl‐prednisolone and taurine on the mdx mouse model of Duchenne muscular dystrophy , 2011, Neuropathology and applied neurobiology.
[3] H. Moulton,et al. Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy. , 2010, Biochimica et biophysica acta.
[4] A. Nakamura,et al. In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse. , 2010, Molecular therapy : the journal of the American Society of Gene Therapy.
[5] C. Reggiani,et al. Oxidative stress by monoamine oxidases is causally involved in myofiber damage in muscular dystrophy. , 2010, Human molecular genetics.
[6] T. Zimmers,et al. Regulation of muscle mass by follistatin and activins. , 2010, Molecular endocrinology.
[7] H. Moulton,et al. Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO. , 2010, Molecular therapy : the journal of the American Society of Gene Therapy.
[8] M. Wood,et al. Identification of a novel muscle targeting peptide in mdx mice , 2010, Peptides.
[9] J. Lachey,et al. Administration of a soluble activin type IIB receptor promotes skeletal muscle growth independent of fiber type. , 2010, Journal of applied physiology.
[10] A. Nakamura,et al. Antisense PMO Found in Dystrophic Dog Model Was Effective in Cells from Exon 7-Deleted DMD Patient , 2010, PloS one.
[11] E. Yeung,et al. IGF-IEc expression, regulation and biological function in different tissues. , 2010, Growth hormone & IGF research : official journal of the Growth Hormone Research Society and the International IGF Research Society.
[12] C. Rapezzi,et al. Antisense modulation of both exonic and intronic splicing motifs induces skipping of a DMD pseudo-exon responsible for x-linked dilated cardiomyopathy. , 2010, Human gene therapy.
[13] S. Bhatnagar,et al. Matrix metalloproteinase inhibitor batimastat alleviates pathology and improves skeletal muscle function in dystrophin-deficient mdx mice. , 2010, The American journal of pathology.
[14] R. Finkel. Read-Through Strategies for Suppression of Nonsense Mutations in Duchenne/ Becker Muscular Dystrophy: Aminoglycosides and Ataluren (PTC124) , 2010, Journal of child neurology.
[15] J. Mendell,et al. Gentamicin‐induced readthrough of stop codons in duchenne muscular dystrophy , 2010, Annals of neurology.
[16] W. Grody,et al. Arginine Metabolism by Macrophages Promotes Cardiac and Muscle Fibrosis in mdx Muscular Dystrophy , 2010, PloS one.
[17] R. Vossen,et al. Accurate quantification of dystrophin mRNA and exon skipping levels in duchenne muscular dystrophy. , 2010, Laboratory investigation; a journal of technical methods and pathology.
[18] G. van Ommen,et al. Exonic sequences provide better targets for antisense oligonucleotides than splice site sequences in the modulation of Duchenne muscular dystrophy splicing. , 2010, Oligonucleotides.
[19] G. Butler-Browne,et al. Combination of myostatin pathway interference and dystrophin rescue enhances tetanic and specific force in dystrophic mdx mice. , 2010, Molecular therapy : the journal of the American Society of Gene Therapy.
[20] M. Wood,et al. Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse. , 2010, Molecular therapy : the journal of the American Society of Gene Therapy.
[21] P. Camelliti,et al. In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy , 2010, The journal of gene medicine.
[22] J. Kornegay,et al. Chronic administration of membrane sealant prevents severe cardiac injury and ventricular dilatation in dystrophic dogs. , 2010, The Journal of clinical investigation.
[23] S. Pandya,et al. Use of Corticosteroids in a Population-Based Cohort of Boys With Duchenne and Becker Muscular Dystrophy , 2010, Journal of child neurology.
[24] Anton Simeonov,et al. Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124 , 2010, Proceedings of the National Academy of Sciences.
[25] Steve D Wilton,et al. Splice modification to restore functional dystrophin synthesis in Duchenne muscular dystrophy. , 2010, Current pharmaceutical design.
[26] P. Sazani,et al. Safety Pharmacology and Genotoxicity Evaluation of AVI-4658 , 2010, International journal of toxicology.
[27] C. Bennett,et al. RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. , 2010, Annual review of pharmacology and toxicology.
[28] K. Bushby,et al. Interventions for muscular dystrophy: molecular medicines entering the clinic , 2009, The Lancet.
[29] F. Muntoni,et al. Immunohistological intensity measurements as a tool to assess sarcolemma‐associated protein expression , 2009, Neuropathology and applied neurobiology.
[30] H. Moulton,et al. A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice. , 2009, Human molecular genetics.
[31] A. Ferlini,et al. Exon skipping‐mediated dystrophin reading frame restoration for small mutations , 2009, Human mutation.
[32] I. Graham,et al. 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 , 2009, The Lancet. Neurology.
[33] Stanley F Nelson,et al. Emerging genetic therapies to treat Duchenne muscular dystrophy , 2009, Current opinion in neurology.
[34] H. Vandenburgh,et al. Automated drug screening with contractile muscle tissue engineered from dystrophic myoblasts , 2009, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.
[35] Jason B. Nikas,et al. Effects of prednisolone on skeletal muscle contractility in mdx mice , 2009, Muscle & nerve.
[36] F. Muntoni,et al. Rational design of antisense oligomers to induce dystrophin exon skipping. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.
[37] S. Bhatnagar,et al. Matrix metalloproteinase-9 inhibition ameliorates pathogenesis and improves skeletal muscle regeneration in muscular dystrophy. , 2009, Human molecular genetics.
[38] I. Graham,et al. Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice. , 2009, Human gene therapy.
[39] Akinori Nakamura,et al. Efficacy of systemic morpholino exon‐skipping in duchenne dystrophy dogs , 2009, Annals of neurology.
[40] J. Schacht,et al. Development of novel aminoglycoside (NB54) with reduced toxicity and enhanced suppression of disease-causing premature stop mutations. , 2009, Journal of medicinal chemistry.
[41] G. MacGowan,et al. Contrasting effects of steroids and angiotensin‐converting‐enzyme inhibitors in a mouse model of dystrophin‐deficient cardiomyopathy , 2009, European journal of heart failure.
[42] O. Dorchies,et al. Protection of dystrophic muscle cells with polyphenols from green tea correlates with improved glutathione balance and increased expression of 67LR, a receptor for (−)‐epigallocatechin gallate , 2009, BioFactors.
[43] Luciano Merlini,et al. Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouse. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.
[44] P. Morcos,et al. Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.
[45] G. S. Pandey,et al. Preclinical drug trials in the mdx mouse: Assessment of reliable and sensitive outcome measures , 2009, Muscle & nerve.
[46] R. Kole,et al. Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers. , 2010, Cardiovascular research.
[47] C. Béroud,et al. Human Splicing Finder: an online bioinformatics tool to predict splicing signals , 2009, Nucleic acids research.
[48] C. Lorson,et al. Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy. , 2009, Human molecular genetics.
[49] S. Sen,et al. Polymersome delivery of siRNA and antisense oligonucleotides. , 2009, Journal of controlled release : official journal of the Controlled Release Society.
[50] D. Auld,et al. Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression , 2009, Proceedings of the National Academy of Sciences.
[51] J. Mendell,et al. Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease , 2009, Muscle & nerve.
[52] J. Chamberlain,et al. Cardiac consequences to skeletal muscle-centric therapeutics for Duchenne muscular dystrophy. , 2009, Trends in cardiovascular medicine.
[53] E. Jackson. The first use of penicillin in the United States. , 2009, Annals of internal medicine.
[54] P. Iversen,et al. By‐passing the nonsense mutation in the 4CV mouse model of muscular dystrophy by induced exon skipping , 2009, The journal of gene medicine.
[55] P. Morcos,et al. Vivo-Morpholinos: a non-peptide transporter delivers Morpholinos into a wide array of mouse tissues. , 2008, BioTechniques.
[56] M. Zatz,et al. Ringo, a Golden Retriever Muscular Dystrophy (GRMD) dog with absent dystrophin but normal strength , 2008, Neuromuscular Disorders.
[57] J. Schertzer,et al. Insulin‐like growth factor‐I analogue protects muscles of dystrophic mdx mice from contraction‐mediated damage , 2008, Experimental physiology.
[58] B. Lebleu,et al. Improved cell-penetrating peptide–PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle , 2008, Nucleic acids research.
[59] N. Almstead,et al. Reading through premature stop codons with PTC1 24. Project Catalyst to find more Duchenne drugs. Interview by Guenter Scheuerbrandt. , 2008, Acta myologica.
[60] J. D’hooge,et al. Long-term blinded placebo-controlled study of SNT-MC17/idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performance , 2008, European heart journal.
[61] P. Iversen,et al. Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. , 2008, Molecular therapy : the journal of the American Society of Gene Therapy.
[62] G. Lutz,et al. Functionalized PEG-PEI copolymers complexed to exon-skipping oligonucleotides improve dystrophin expression in mdx mice. , 2008, Human gene therapy.
[63] C. Reggiani,et al. In vivo delivery of naked antisense oligos in aged mdx mice: Analysis of dystrophin restoration in skeletal and cardiac muscle , 2008, Neuromuscular Disorders.
[64] M. Grounds,et al. Towards developing standard operating procedures for pre-clinical testing in the mdx mouse model of Duchenne muscular dystrophy , 2008, Neurobiology of Disease.
[65] K. Wee,et al. Dynamics of Co-Transcriptional Pre-mRNA Folding Influences the Induction of Dystrophin Exon Skipping by Antisense Oligonucleotides , 2008, PloS one.
[66] J. Mendell,et al. Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors , 2008, Proceedings of the National Academy of Sciences.
[67] K. Tsuchida,et al. Transgenic expression of a myostatin inhibitor derived from follistatin increases skeletal muscle mass and ameliorates dystrophic pathology in mdx mice , 2008, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.
[68] Johan T den Dunnen,et al. Local dystrophin restoration with antisense oligonucleotide PRO051. , 2007, The New England journal of medicine.
[69] N. Darín,et al. Inflammation and response to steroid treatment in limb-girdle muscular dystrophy 2I. , 2007, European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society.
[70] I. Graham,et al. Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle. , 2007, Human gene therapy.
[71] C. Angelini. The role of corticosteroids in muscular dystrophy: A critical appraisal , 2007, Muscle & nerve.
[72] P. Iversen,et al. Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse. , 2007, Molecular therapy : the journal of the American Society of Gene Therapy.
[73] T. Südhof,et al. Primary Role of Functional Ischemia, Quantitative Evidence for the Two-Hit Mechanism, and Phosphodiesterase-5 Inhibitor Therapy in Mouse Muscular Dystrophy , 2007, PloS one.
[74] N. Corbi,et al. Utrophin Up-Regulation by an Artificial Transcription Factor in Transgenic Mice , 2007, PloS one.
[75] D. A. Stein,et al. Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo. , 2007, Biochemical Society transactions.
[76] P. Iversen,et al. Bmc Molecular Biology Antisense Oligonucleotide Induced Exon Skipping and the Dystrophin Gene Transcript: Cocktails and Chemistries , 2022 .
[77] S. Wilton,et al. Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript. , 2007, Molecular therapy : the journal of the American Society of Gene Therapy.
[78] Meenal Patel,et al. PTC124 targets genetic disorders caused by nonsense mutations , 2007, Nature.
[79] S. Peltz,et al. Safety, Tolerability, and Pharmacokinetics of PTC124, a Nonaminoglycoside Nonsense Mutation Suppressor, Following Single‐ and Multiple‐Dose Administration to Healthy Male and Female Adult Volunteers , 2007, Journal of clinical pharmacology.
[80] J. Metzger,et al. Cardiomyopathy of Duchenne muscular dystrophy: pathogenesis and prospect of membrane sealants as a new therapeutic approach , 2007, Expert review of cardiovascular therapy.
[81] N. F. Kamel,et al. The effects of glucocorticoid therapy on the inflammatory and Dendritic cells in muscular dystrophies , 2006, International journal of experimental pathology.
[82] S. Hamed. Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy. , 2006, IDrugs : the investigational drugs journal.
[83] G. van Ommen,et al. Entries in the Leiden Duchenne muscular dystrophy mutation database: An overview of mutation types and paradoxical cases that confirm the reading‐frame rule , 2006, Muscle & nerve.
[84] Y. Takeshima,et al. Intravenous Infusion of an Antisense Oligonucleotide Results in Exon Skipping in Muscle Dystrophin mRNA of Duchenne Muscular Dystrophy , 2006, Pediatric Research.
[85] B. Alman,et al. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade , 2006, Neuromuscular Disorders.
[86] P. Miura,et al. Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: how close are we? , 2006, Trends in molecular medicine.
[87] A. Rabinowitz,et al. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology , 2006, Nature Medicine.
[88] S. Wilton,et al. Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide , 2006, The journal of gene medicine.
[89] M. Tarnopolsky,et al. Nutritional therapy improves function and complements corticosteroid intervention in mdx mice , 2006, Muscle & nerve.
[90] D. A. Stein,et al. Gene-Specific Countermeasures against Ebola Virus Based on Antisense Phosphorodiamidate Morpholino Oligomers , 2006, PLoS pathogens.
[91] G. van Ommen,et al. Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites. , 2005, Oligonucleotides.
[92] K. Davies,et al. Utrophin upregulation in Duchenne muscular dystrophy. , 2005, Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology.
[93] D. A. Stein,et al. Antiviral activity of morpholino oligomers designed to block various aspects of Equine arteritis virus amplification in cell culture. , 2005, The Journal of general virology.
[94] G. Lutz,et al. Poly(ethylene imine)-poly(ethylene glycol) copolymers facilitate efficient delivery of antisense oligonucleotides to nuclei of mature muscle cells of mdx mice. , 2005, Human gene therapy.
[95] Y. Takeshima,et al. Intraperitoneal administration of phosphorothioate antisense oligodeoxynucleotide against splicing enhancer sequence induced exon skipping in dystrophin mRNA expressed in mdx skeletal muscle , 2005, Brain and Development.
[96] J. Bourke,et al. The multidisciplinary management of Duchenne muscular dystrophy , 2005 .
[97] K. Patel,et al. The function of Myostatin and strategies of Myostatin blockade—new hope for therapies aimed at promoting growth of skeletal muscle , 2005, Neuromuscular Disorders.
[98] A. Rabinowitz,et al. Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. , 2005, Proceedings of the National Academy of Sciences of the United States of America.
[99] N. Kipshidze,et al. Antisense therapy for restenosis following percutaneous coronary intervention , 2005, Expert opinion on biological therapy.
[100] R. Griggs,et al. Report on the 124th ENMC International Workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids 2–4 April 2004, Naarden, The Netherlands , 2004, Neuromuscular Disorders.
[101] G. V. Ommen,et al. Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells , 2004, Gene Therapy.
[102] B. Alman,et al. Deflazacort in Duchenne muscular dystrophy: a comparison of two different protocols , 2004, Neuromuscular Disorders.
[103] Y. Takeshima,et al. Chimeric RNA/ethylene-bridged nucleic acids promote dystrophin expression in myocytes of duchenne muscular dystrophy by inducing skipping of the nonsense mutation-encoding exon. , 2004, Human gene therapy.
[104] R. Vossen,et al. Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides. , 2004, Molecular therapy : the journal of the American Society of Gene Therapy.
[105] K. Tsuchida. Activins, myostatin and related TGF-beta family members as novel therapeutic targets for endocrine, metabolic and immune disorders. , 2004, Current drug targets. Immune, endocrine and metabolic disorders.
[106] S. Wilton,et al. Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle. , 2003, Human molecular genetics.
[107] C. Mann,et al. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse , 2003, Nature Medicine.
[108] Jinhua Wang,et al. ESEfinder: a web resource to identify exonic splicing enhancers , 2003, Nucleic Acids Res..
[109] P. Iversen,et al. Efficacy of antisense morpholino oligomer targeted to c-myc in prostate cancer xenograft murine model and a Phase I safety study in humans. , 2003, Clinical cancer research : an official journal of the American Association for Cancer Research.
[110] C. Mann,et al. Target selection for antisense oligonucleotide induced exon skipping in the dystrophin gene , 2003, The journal of gene medicine.
[111] C. Silversides,et al. Effects of deflazacort on left ventricular function in patients with Duchenne muscular dystrophy. , 2003, The American journal of cardiology.
[112] A. De Luca,et al. Enhanced Dystrophic Progression in mdx Mice by Exercise and Beneficial Effects of Taurine and Insulin-Like Growth Factor-1 , 2003, Journal of Pharmacology and Experimental Therapeutics.
[113] Colin Chandler,et al. Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation , 2002, Neuromuscular Disorders.
[114] C. Mann,et al. Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy , 2002, The journal of gene medicine.
[115] R. Hewick,et al. The Myostatin Propeptide and the Follistatin-related Gene Are Inhibitory Binding Proteins of Myostatin in Normal Serum* , 2002, The Journal of Biological Chemistry.
[116] G. V. Ommen,et al. Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy , 2002, Neuromuscular Disorders.
[117] A. Nakamura,et al. Progression of dystrophic features and activation of mitogen‐activated protein kinases and calcineurin by physical exercise, in hearts of mdx mice , 2002, FEBS letters.
[118] K. Davies,et al. Non-toxic ubiquitous over-expression of utrophin in the mdx mouse , 2001, Neuromuscular Disorders.
[119] R. Mutani,et al. Late onset and very mild course of Xp21 Becker type muscular dystrophy. , 2001, Clinical neuropathology.
[120] K. Fischbeck,et al. Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations , 2001, Annals of neurology.
[121] C. Mann,et al. Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. , 2001, Proceedings of the National Academy of Sciences of the United States of America.
[122] J. T. Dunnen,et al. Dystrophin nonsense mutation induces different levels of exon 29 skipping and leads to variable phenotypes within one BMD family , 2000, European Journal of Human Genetics.
[123] M. S. Hudecki,et al. Pre-clinical screening of drugs using the mdx mouse , 2000, Neuromuscular Disorders.
[124] S. Wilton,et al. Massive Idiosyncratic Exon Skipping Corrects the Nonsense Mutation in Dystrophic Mouse Muscle and Produces Functional Revertant Fibers by Clonal Expansion , 2000, The Journal of cell biology.
[125] J. Summerton. Morpholino antisense oligomers: the case for an RNase H-independent structural type. , 1999, Biochimica et biophysica acta.
[126] S. Agrawal,et al. Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides , 1999, Neuromuscular Disorders.
[127] K. Davies,et al. Adenovirus-mediated utrophin gene transfer mitigates the dystrophic phenotype of mdx mouse muscles. , 1999, Human gene therapy.
[128] C. Mann,et al. Alternative dystrophin gene transcripts in golden retriever muscular dystrophy , 1998, Muscle & nerve.
[129] M. Manoharan,et al. Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides. , 1998, Human molecular genetics.
[130] K. Davies,et al. Efficient utrophin expression following adenovirus gene transfer in dystrophic muscle. , 1998, Biochemical and biophysical research communications.
[131] J. Clancy,et al. Suppression of a CFTR premature stop mutation in a bronchial epithelial cell line , 1997, Nature Medicine.
[132] K. Davies,et al. Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene , 1996, Nature.
[133] Y. Takeshima,et al. Induction of exon skipping of the dystrophin transcript in lymphoblastoid cells by transfecting an antisense oligodeoxynucleotide complementary to an exon recognition sequence. , 1996, Biochemical and biophysical research communications.
[134] K. Folkers,et al. Two successful double-blind trials with coenzyme Q10 (vitamin Q10) on muscular dystrophies and neurogenic atrophies. , 1995, Biochimica et biophysica acta.
[135] Y. Takeshima,et al. Modulation of in vitro splicing of the upstream intron by modifying an intra-exon sequence which is deleted from the dystrophin gene in dystrophin Kobe. , 1995, The Journal of clinical investigation.
[136] K. Bushby,et al. Becker muscular dystrophy with onset after 60 years , 1994, Neurology.
[137] K. Davies,et al. Dystrophin and dystrophin-related proteins: A review of protein and RNA studies , 1993, Neuromuscular Disorders.
[138] T. Vulliamy,et al. Exon skipping and translation in patients with frameshift deletions in the dystrophin gene. , 1993, American journal of human genetics.
[139] R. Bartlett,et al. An error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophy. , 1992, Genomics.
[140] K. Davies,et al. Very mild muscular dystrophy associated with the deletion of 46% of dystrophin , 1990, Nature.
[141] K. Fischbeck,et al. Familial X‐linked myalgia and cramps , 1989, Neurology.
[142] L. Kunkel,et al. The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion. , 1989, American journal of human genetics.
[143] C. van Broeckhoven,et al. Germinal mosaicism increases the recurrence risk for 'new' Duchenne muscular dystrophy mutations. , 1989, Journal of medical genetics.
[144] E A Barnard,et al. The molecular basis of muscular dystrophy in the mdx mouse: a point mutation. , 1989, Science.
[145] D. Hill,et al. An autosomal transcript in skeletal muscle with homology to dystrophin , 1989, Nature.
[146] Doris M. Miller,et al. Muscular dystrophy in a litter of golden retriever dogs , 1988, Muscle & nerve.
[147] M. Koenig,et al. Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals , 1987, Cell.
[148] J. Burke,et al. Suppression of a nonsense mutation in mammalian cells in vivo by the aminoglycoside antibiotics G-418 and paromomycin. , 1985, Nucleic acids research.
[149] K. Moore,et al. X chromosome-linked muscular dystrophy (mdx) in the mouse. , 1984, Proceedings of the National Academy of Sciences of the United States of America.
[150] H. Moulton,et al. CPP-directed oligonucleotide exon skipping in animal models of Duchenne muscular dystrophy. , 2011, Methods in molecular biology.
[151] Peijuan Lu,et al. Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino , 2010, Gene Therapy.
[152] K. Davies,et al. Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping. , 2010, Molecular therapy : the journal of the American Society of Gene Therapy.
[153] S. Wilton,et al. The influence of antisense oligonucleotide length on dystrophin exon skipping. , 2007, Molecular therapy : the journal of the American Society of Gene Therapy.
[154] A. Manzur,et al. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. , 2004, The Cochrane database of systematic reviews.
[155] Peter Sazani,et al. Effects of base modifications on antisense properties of 2'-O-methoxyethyl and PNA oligonucleotides. , 2003, Antisense & nucleic acid drug development.
[156] D. Wells,et al. Immune responses to dystropin: implications for gene therapy of Duchenne muscular dystrophy. , 2000, Gene therapy.
[157] A. Monaco,et al. An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. , 1988, Genomics.
[158] M. Stephenson,et al. Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide. , 1978, Proceedings of the National Academy of Sciences of the United States of America.