Published in Neuromuscul Disord on July 01, 1999
RNA therapeutics: beyond RNA interference and antisense oligonucleotides. Nat Rev Drug Discov (2012) 3.70
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc Natl Acad Sci U S A (2001) 2.71
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. Mol Ther (2008) 2.69
Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy. J Clin Invest (2007) 2.68
Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion. J Cell Biol (2000) 2.24
A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse. Hum Mol Genet (2011) 2.13
Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing. J Clin Invest (2003) 1.89
Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers. Cardiovasc Res (2009) 1.82
Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications. RNA (2007) 1.78
Duchenne muscular dystrophy and dystrophin: pathogenesis and opportunities for treatment. EMBO Rep (2004) 1.66
Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense. Am J Hum Genet (2003) 1.52
The FSHD atrophic myotube phenotype is caused by DUX4 expression. PLoS One (2011) 1.45
Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment. Mol Ther Nucleic Acids (2012) 1.41
In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse. Mol Ther (2010) 1.33
Therapeutic potential of splice-switching oligonucleotides. Oligonucleotides (2009) 1.24
Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells. Proc Natl Acad Sci U S A (2002) 1.24
Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients. Proc Natl Acad Sci U S A (2000) 1.22
RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice. Proc Natl Acad Sci U S A (2009) 1.21
Gene and cell-mediated therapies for muscular dystrophy. Muscle Nerve (2013) 1.14
Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries. BMC Mol Biol (2007) 1.11
Dynamics of co-transcriptional pre-mRNA folding influences the induction of dystrophin exon skipping by antisense oligonucleotides. PLoS One (2008) 1.09
Gene therapy in large animal models of muscular dystrophy. ILAR J (2009) 1.05
Induction of revertant fibres in the mdx mouse using antisense oligonucleotides. Genet Vaccines Ther (2006) 0.95
Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy. Ther Adv Neurol Disord (2010) 0.93
Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches. Nucleic Acids Res (2013) 0.91
Nanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of mdx mice. BMC Biotechnol (2008) 0.90
Targeted gene repair -- in the arena. J Clin Invest (2003) 0.90
Temperature-dependent splicing of beta-globin pre-mRNA. Nucleic Acids Res (2002) 0.90
Therapeutic exon skipping for dysferlinopathies? Eur J Hum Genet (2010) 0.89
Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy. Biomed Res Int (2013) 0.88
Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells. Nucleic Acids Res (2013) 0.86
Effective exon skipping and dystrophin restoration by 2'-o-methoxyethyl antisense oligonucleotide in dystrophin-deficient mice. PLoS One (2013) 0.86
Molecular diagnosis of duchenne muscular dystrophy: past, present and future in relation to implementing therapies. Clin Biochem Rev (2011) 0.83
The potential of exon skipping for treatment for Duchenne muscular dystrophy. J Child Neurol (2010) 0.81
Antisense mediated splicing modulation for inherited metabolic diseases: challenges for delivery. Nucleic Acid Ther (2014) 0.81
Ameliorating pathogenesis by removing an exon containing a missense mutation: a potential exon-skipping therapy for laminopathies. Gene Ther (2015) 0.80
Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy. Sci Rep (2015) 0.79
Directed Alternative Splicing in Nijmegen Breakage Syndrome: Proof of Principle Concerning Its Therapeutical Application. Mol Ther (2015) 0.77
Antisense oligonucleotide-based therapy in human erythropoietic protoporphyria. Am J Hum Genet (2014) 0.77
Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides. Mol Ther Nucleic Acids (2014) 0.77
High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics. Biology (Basel) (2014) 0.75
Genetic correction of splice site mutation in purified and enriched myoblasts isolated from mdx5cv mice. BMC Mol Biol (2009) 0.75
Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents. Appl Clin Genet (2011) 0.75
A New Kid on the Playground of CRISPR DMD Therapy. Hum Gene Ther Clin Dev (2017) 0.75
Long-term outcome after intravenous magnesium sulphate in suspected acute myocardial infarction: the second Leicester Intravenous Magnesium Intervention Trial (LIMIT-2) Lancet (1994) 8.63
Detection of nucleic acid hybridization by nonradiative fluorescence resonance energy transfer. Proc Natl Acad Sci U S A (1988) 4.90
Retracted First myocardial infarction in patients of Indian subcontinent and European origin: comparison of risk factors, management, and long term outcome. BMJ (1997) 4.24
Efficient methods for attaching non-radioactive labels to the 5' ends of synthetic oligodeoxyribonucleotides. Nucleic Acids Res (1986) 4.12
Sequence identity of the n-1 product of a synthetic oligonucleotide. Nucleic Acids Res (1995) 3.88
Ribonuclease P: an enzyme with an essential RNA component. Proc Natl Acad Sci U S A (1978) 3.52
Intravenous magnesium sulphate in suspected acute myocardial infarction: results of the second Leicester Intravenous Magnesium Intervention Trial (LIMIT-2) Lancet (1992) 3.40
Selection of splice sites in pre-mRNAs with short internal exons. Mol Cell Biol (1991) 3.02
Inhibition of human immunodeficiency virus replication by antisense oligodeoxynucleotides. Proc Natl Acad Sci U S A (1988) 2.85
Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides. Proc Natl Acad Sci U S A (1993) 2.79
SCH 503034, a mechanism-based inhibitor of hepatitis C virus NS3 protease, suppresses polyprotein maturation and enhances the antiviral activity of alpha interferon in replicon cells. Antimicrob Agents Chemother (2006) 2.75
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc Natl Acad Sci U S A (2001) 2.71
Arterial versus capillary sampling for analysing blood gas pressures. BMJ (1995) 2.55
Antibodies to hnRNP core proteins inhibit in vitro splicing of human beta-globin pre-mRNA. Nucleic Acids Res (1986) 2.28
Selection of novel exon recognition elements from a pool of random sequences. Mol Cell Biol (1995) 2.27
Older adults and withdrawal from benzodiazepine hypnotics in general practice: effects on cognitive function, sleep, mood and quality of life. Psychol Med (2003) 2.17
Inhibition of acquired immunodeficiency syndrome virus by oligodeoxynucleoside methylphosphonates. Proc Natl Acad Sci U S A (1988) 2.09
Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. Gene Ther (2006) 2.00
Laparoscopic assisted transversus abdominis plane block: a novel insertion technique during laparoscopic nephrectomy. Anaesthesia (2011) 1.98
Rapid correction of low vitamin D status in nursing home residents. Osteoporos Int (2008) 1.97
Barriers to treatment: why alcohol and drug abusers delay or never seek treatment. Addict Behav (1993) 1.93
Acute hepatitis associated with the use of a Chinese herbal product, ma-huang. Am J Gastroenterol (1996) 1.87
Acute small-bowel obstruction following mesenteric perforation by CAPD catheter. Nephrol Dial Transplant (1997) 1.85
E. coli RNAase P has a required RNA component. Cell (1980) 1.80
Accurate in vitro splicing of human beta-globin RNA. Nucleic Acids Res (1982) 1.73
Cooperation of pre-mRNA sequence elements in splice site selection. Mol Cell Biol (1992) 1.70
The length of the downstream exon and the substitution of specific sequences affect pre-mRNA splicing in vitro. Mol Cell Biol (1988) 1.69
Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs. Nucleic Acids Res (2001) 1.67
A prospective study of conventional transbronchial needle aspiration: performance and cost utility. Respiration (2010) 1.66
Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development. Biochemistry (1998) 1.66
Humoral and cellular immune responses up to 7.5 years after administration of streptokinase for acute myocardial infarction. Eur Heart J (1999) 1.64
Gene mapping in Gypsies identifies a novel demyelinating neuropathy on chromosome 8q24. Nat Genet (1996) 1.61
Specific inhibition of human immunodeficiency virus type 1 replication by antisense oligonucleotides: an in vitro model for treatment. Proc Natl Acad Sci U S A (1992) 1.59
Local anaesthesia: to warm or alter the pH? A survey of current practice. J R Coll Surg Edinb (1999) 1.58
Repair of thalassemic human beta-globin mRNA in mammalian cells by antisense oligonucleotides. Proc Natl Acad Sci U S A (1996) 1.58
Quality control of anti-tuberculosis fixed-dose combination formulations in the global market: an in vitro study. Int J Tuberc Lung Dis (2004) 1.58
The association between narcolepsy and REM behavior disorder (RBD). Sleep Med (2005) 1.56
Are patients who undergo open-access endoscopy more anxious about their procedures than patients referred from the GI clinic? Am J Gastroenterol (1996) 1.53
Self-stabilized antisense oligodeoxynucleotide phosphorothioates: properties and anti-HIV activity. Nucleic Acids Res (1993) 1.53
Cross-cultural evaluation of two drinking assessment instruments: alcohol timeline followback and inventory of drinking situations. Subst Use Misuse (2001) 1.53
RNase H cleavage of RNA hybridized to oligonucleotides containing methylphosphonate, phosphorothioate and phosphodiester bonds. Nucleic Acids Res (1989) 1.52
Inhibition of splicing but not cleavage at the 5' splice site by truncating human beta-globin pre-mRNA. Proc Natl Acad Sci U S A (1986) 1.51
Comparative pharmacology of epibatidine: a potent agonist for neuronal nicotinic acetylcholine receptors. Mol Pharmacol (1995) 1.49
Ocular myocysticercosis: an unusual case of ptosis. Nepal J Ophthalmol (2013) 1.47
Stability of antisense DNA oligodeoxynucleotide analogs in cellular extracts and sera. Life Sci (1991) 1.46
GEM 91--an antisense oligonucleotide phosphorothioate as a therapeutic agent for AIDS. Antisense Res Dev (1992) 1.45
Use of parenteral methotrexate significantly reduces the need for biological therapy. Rheumatology (Oxford) (2007) 1.43
Use of transillumination technique for venous cannulation in paediatric patients under anaesthesia. Singapore Med J (2010) 1.41
Modification of alternative splicing of Bcl-x pre-mRNA in prostate and breast cancer cells. analysis of apoptosis and cell death. J Biol Chem (2001) 1.40
The in vitro-synthesized RNA from a cDNA clone of hepatitis E virus is infectious. J Virol (2000) 1.40
Bilateral retinopathy, aplastic anaemia, and central nervous system abnormalities: a new syndrome? J Med Genet (1992) 1.40
Zosteriform and disseminated lesions in cutaneous leiomyoma. Acta Derm Venereol (2001) 1.40
Half a loaf is better than waiting for the bread truck. A computerized mini-medical record for outpatient care. Arch Intern Med (1992) 1.39
Reconstitution of RNase P activity from inactive RNA and protein. Proc Natl Acad Sci U S A (1979) 1.39
Extracorporeal whole body hyperthermia treatment of HIV patients, a feasibility study. Int J Hyperthermia (1998) 1.38
Percutaneous treatment of catheter-induced dissection of the left main coronary artery and adjacent aortic wall: a case report. Catheter Cardiovasc Interv (2000) 1.35
Sequences of human repetitive DNA, non-alpha-globin genes, and major histocompatibility locus genes. Cold Spring Harb Symp Quant Biol (1983) 1.33
The influence of antisense oligonucleotide length on dystrophin exon skipping. Mol Ther (2007) 1.30
Synergistic activation of p53 by inhibition of MDM2 expression and DNA damage. Proc Natl Acad Sci U S A (1998) 1.30
A comparison of a brief and long version of the Situational Confidence Questionnaire. Behav Res Ther (2000) 1.27
Alcohol abusers who want to quit smoking: implications for clinical treatment. Drug Alcohol Depend (1999) 1.27
Identification of exon sequences involved in splice site selection. J Biol Chem (1994) 1.26
Histopathology of papilloma virus infection of the cervix uteri: the history, taxonomy, nomenclature and reporting of koilocytic dysplasias. J Clin Pathol (1983) 1.23
Survivin inhibition induces human neural tumor cell death through caspase-independent and -dependent pathways. J Neurochem (2001) 1.23
Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients. Proc Natl Acad Sci U S A (2000) 1.22
Correction of aberrant splicing of the cystic fibrosis transmembrane conductance regulator (CFTR) gene by antisense oligonucleotides. J Biol Chem (1999) 1.22
Autosomal dominant distal myopathy: linkage to chromosome 14. Am J Hum Genet (1995) 1.22
Properties of purified ribonuclease P from Escherichia coli. Biochemistry (1981) 1.22
[3H]L-655,708, a novel ligand selective for the benzodiazepine site of GABAA receptors which contain the alpha 5 subunit. Neuropharmacology (1996) 1.21
Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs. Proc Natl Acad Sci U S A (1998) 1.20
A splicing factor that is inactivated during in vivo heat shock is functionally equivalent to the [U4/U6.U5] triple snRNP-specific proteins. Genes Dev (1992) 1.20
Dual blockade of cyclic AMP response element- (CRE) and AP-1-directed transcription by CRE-transcription factor decoy oligonucleotide. gene-specific inhibition of tumor growth. J Biol Chem (1999) 1.20
Long-term treatment of human immunodeficiency virus-infected cells with antisense oligonucleotide phosphorothioates. Proc Natl Acad Sci U S A (1993) 1.19
Large-scale synthesis, purification, and analysis of oligodeoxynucleotide phosphorothioates. Antisense Res Dev (1994) 1.17
Modification of alternative splicing pathways as a potential approach to chemotherapy. Pharmacol Ther (2000) 1.17
Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo. Biochem Soc Trans (2007) 1.17
Translation of chick aortic elastin messenger ribonucleic acid. Comparison to elastin synthesis in chick aorta organ culture. Biochemistry (1980) 1.16
Heat shock protein 70 prevents secretagogue-induced cell injury in the pancreas by preventing intracellular trypsinogen activation. J Clin Invest (2000) 1.15
Modification of alternative splicing by antisense oligonucleotides as a potential chemotherapy for cancer and other diseases. Curr Cancer Drug Targets (2001) 1.15
Complement activation and hemodynamic changes following intravenous administration of phosphorothioate oligonucleotides in the monkey. Antisense Res Dev (1994) 1.14
Significance of chimerism in hematopoietic stem cell transplantation: new variations on an old theme. Bone Marrow Transplant (2004) 1.13
In vivo studies with antisense oligonucleotides. Trends Pharmacol Sci (1997) 1.12
TM Finder: a prediction program for transmembrane protein segments using a combination of hydrophobicity and nonpolar phase helicity scales. Protein Sci (2001) 1.12
Antisense anti-MDM2 oligonucleotides as a novel therapeutic approach to human breast cancer: in vitro and in vivo activities and mechanisms. Clin Cancer Res (2001) 1.12
Cloning, sequencing, and expression of the hepatitis E virus (HEV) nonstructural open reading frame 1 (ORF1). J Med Virol (2000) 1.12
A common human beta globin splicing mutation modeled in mice. Blood (1998) 1.11
Attitudes to long-term use of benzodiazepine hypnotics by older people in general practice: findings from interviews with service users and providers. Aging Ment Health (2004) 1.10
Antitumor activity and pharmacokinetics of a mixed-backbone antisense oligonucleotide targeted to the RIalpha subunit of protein kinase A after oral administration. Proc Natl Acad Sci U S A (1999) 1.09
Mutation segregation and rapid carrier detection of X-linked muscular dystrophy in dogs. Am J Vet Res (1996) 1.09
Identification and characterization by antisense oligonucleotides of exon and intron sequences required for splicing. Mol Cell Biol (1994) 1.08
Pattern and kinetics of cytokine production following administration of phosphorothioate oligonucleotides in mice. Antisense Nucleic Acid Drug Dev (1997) 1.08
Inactivation of splicing factors in HeLa cells subjected to heat shock. J Biol Chem (1990) 1.08
Retinal complications associated with hyperviscosity in chronic lymphocytic leukaemia. Intern Med J (2008) 1.08
Neuropeptides modulate immune deviation induced via the anterior chamber of the eye. J Immunol (1995) 1.08