The potential of antisense oligonucleotide therapies for inherited childhood lung diseases
暂无分享,去创建一个
Sue Fletcher | K. Martinovich | S. Stick | A. Kicic | S. Wilton | S. Fletcher | A. Schultz | Kelly M. Martinovich | Nicole C. Shaw | Anthony Kicic | André Schultz | Steve D. Wilton | Stephen M. Stick | N. Shaw
[1] Antisense oligonucleotides in cancer , 2014, Current opinion in oncology.
[2] M. Welsh,et al. Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis , 1993, Cell.
[3] R. Kole,et al. Modification of alternative splicing by antisense therapeutics. , 2004, Oligonucleotides.
[4] C. Lorson,et al. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy. , 1999, Proceedings of the National Academy of Sciences of the United States of America.
[5] Timothy Sterne-Weiler,et al. Exon identity crisis: disease-causing mutations that disrupt the splicing code , 2014, Genome Biology.
[6] C. Wallis,et al. Ten‐year follow up of hydroxychloroquine treatment for ABCA3 deficiency , 2014, Pediatric pulmonology.
[7] Thomas Jaki,et al. A Bayesian adaptive design for clinical trials in rare diseases , 2016, Comput. Stat. Data Anal..
[8] H. H. Choo,et al. Inhaled p38alpha mitogen-activated protein kinase antisense oligonucleotide attenuates asthma in mice. , 2005, American journal of respiratory and critical care medicine.
[9] Daniel G. Anderson,et al. Non-viral vectors for gene-based therapy , 2014, Nature Reviews Genetics.
[10] J. McDonald,et al. Local and systemic tolerability of a 2′O-methoxyethyl antisense oligonucleotide targeting interleukin-4 receptor-α delivery by inhalation in mouse and monkey , 2014, Inhalation toxicology.
[11] Christopher E. Hart,et al. Integrated Safety Assessment of 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human Volunteers , 2016, Molecular therapy : the journal of the American Society of Gene Therapy.
[12] K. Flanigan,et al. Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene , 2014, Molecular therapy. Nucleic acids.
[13] S. Takeda,et al. Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy , 2016, Journal of visualized experiments : JoVE.
[14] H. Gourabi,et al. Molecular study of (TG)m(T)n polymorphisms in Iranian males with congenital bilateral absence of the vas deferens. , 2007, Journal of andrology.
[15] S. Lai,et al. Evading immune cell uptake and clearance requires PEG grafting at densities substantially exceeding the minimum for brush conformation. , 2014, Molecular pharmaceutics.
[16] B. Roehr. Fomivirsen approved for CMV retinitis. , 1998, Journal of the International Association of Physicians in AIDS Care.
[17] J. Moss,et al. Variable deletion of exon 9 coding sequences in cystic fibrosis transmembrane conductance regulator gene mRNA transcripts in normal bronchial epithelium. , 1991, The EMBO journal.
[18] J. Karras,et al. Inhaled CD86 Antisense Oligonucleotide Suppresses Pulmonary Inflammation and Airway Hyper-Responsiveness in Allergic Mice , 2007, Journal of Pharmacology and Experimental Therapeutics.
[19] C. Mann,et al. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse , 2003, Nature Medicine.
[20] W. V. van Roon-Mom,et al. Antisense oligonucleotides in therapy for neurodegenerative disorders. , 2015, Advanced drug delivery reviews.
[21] H. Cantiello,et al. Reversal of cystic fibrosis phenotype in a cultured Delta508 cystic fibrosis transmembrane conductance regulator cell line by oligonucleotide insertion. , 2004, Proceedings of the National Academy of Sciences of the United States of America.
[22] N. Dias,et al. Antisense oligonucleotides: basic concepts and mechanisms. , 2002, Molecular cancer therapeutics.
[23] J C Olsen,et al. A novel mutation in the cystic fibrosis gene in patients with pulmonary disease but normal sweat chloride concentrations. , 1994, The New England journal of medicine.
[24] D. Corey,et al. Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs , 2017, Nucleic acids research.
[25] L. Nogee. Abnormal expression of surfactant protein C and lung disease. , 2002, American journal of respiratory cell and molecular biology.
[26] A. Hamvas,et al. An intronic ABCA3 mutation responsible for respiratory disease , 2012, Pediatric Research.
[27] F. Goñi,et al. Lipid-modified oligonucleotide conjugates: Insights into gene silencing, interaction with model membranes and cellular uptake mechanisms. , 2017, Bioorganic & medicinal chemistry.
[28] Z. Dominski,et al. Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides. , 1993, Proceedings of the National Academy of Sciences of the United States of America.
[29] J. Weissenbach,et al. Identification and characterization of a spinal muscular atrophy-determining gene , 1995, Cell.
[30] M. Stephenson,et al. Inhibition of Rous sarcoma viral RNA translation by a specific oligodeoxyribonucleotide. , 1978, Proceedings of the National Academy of Sciences of the United States of America.
[31] J. Karras,et al. Anti-Inflammatory Activity of Inhaled IL-4 Receptor-α Antisense Oligonucleotide in Mice , 2007 .
[32] Mallory A. Havens,et al. Splice-switching antisense oligonucleotides as therapeutic drugs , 2016, Nucleic acids research.
[33] M. Wood,et al. Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice , 2016, Nature Communications.
[34] Elaine Wong,et al. Mipomersen (kynamro): a novel antisense oligonucleotide inhibitor for the management of homozygous familial hypercholesterolemia. , 2014, P & T : a peer-reviewed journal for formulary management.
[35] R. Finkel,et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study , 2016, The Lancet.
[36] L. Nogee,et al. Surfactant dysfunction. , 2011, Paediatric respiratory reviews.
[37] A. Bush,et al. ABCA3 transporter deficiency. , 2012, American journal of respiratory and critical care medicine.
[38] W. Finkbeiner,et al. In vivo and in vitro ivacaftor response in cystic fibrosis patients with residual CFTR function: N‐of‐1 studies , 2017, Pediatric pulmonology.
[39] C. Bennett,et al. Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides. , 2015, Advanced drug delivery reviews.
[40] N. Aronin,et al. Huntingtin‐lowering strategies in Huntington's disease: Antisense oligonucleotides, small RNAs, and gene editing , 2014, Movement disorders : official journal of the Movement Disorder Society.
[41] J. Goldblatt,et al. Intron-8 polythymidine sequence in Australasian individuals with CF mutations R117H and R117C. , 2001, The European respiratory journal.
[42] S. Russo,et al. Biosynthesis of Surfactant Protein C (SP-C) , 2002, The Journal of Biological Chemistry.
[43] J. Whitsett,et al. Genetic Disorders of Surfactant Dysfunction , 2009, Pediatric and developmental pathology : the official journal of the Society for Pediatric Pathology and the Paediatric Pathology Society.
[44] D. Guyer,et al. Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease , 2006, Nature Reviews Drug Discovery.
[45] P. Nielsen,et al. In vitro transcriptional and translational block of the bcl-2 gene operated by peptide nucleic acid. , 1999, Biochemical and biophysical research communications.
[46] Xiaoyuan Chen,et al. Intracellular delivery of an anionic antisense oligonucleotide via receptor-mediated endocytosis , 2008, Nucleic acids research.
[47] S. Russo,et al. A surfactant protein C precursor protein BRICHOS domain mutation causes endoplasmic reticulum stress, proteasome dysfunction, and caspase 3 activation. , 2005, American journal of respiratory cell and molecular biology.
[48] J. Mendell,et al. Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History , 2017, Journal of neuromuscular diseases.
[49] G. Stone,et al. Intramural coronary delivery of advanced antisense oligonucleotides reduces neointimal formation in the porcine stent restenosis model. , 2002, Journal of the American College of Cardiology.
[50] A. Pêgo,et al. Therapeutic antisense oligonucleotides against cancer: hurdling to the clinic , 2014, Front. Chem..
[51] B. Wong,et al. Multiple exon skipping strategies to by-pass dystrophin mutations , 2012, Neuromuscular Disorders.
[52] Heinz Schmidli,et al. Meta-analytic-predictive use of historical variance data for the design and analysis of clinical trials , 2017, Comput. Stat. Data Anal..
[53] M. Griese. Pulmonary surfactant in health and human lung diseases: state of the art. , 1999, The European respiratory journal.
[54] S. Feng,et al. Nanocarriers for delivery of siRNA and co-delivery of siRNA and other therapeutic agents. , 2015, Nanomedicine.
[55] J. Karras,et al. Anti-inflammatory activity of inhaled IL-4 receptor-alpha antisense oligonucleotide in mice. , 2007, American journal of respiratory cell and molecular biology.
[56] J. Clancy,et al. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. , 2010, The New England journal of medicine.
[57] A. Hamvas,et al. Lung Transplantation for Inherited Disorders of Surfactant Metabolism , 2008 .
[58] Jinglan Zhou,et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770 , 2009, Proceedings of the National Academy of Sciences.
[59] D. Paton. Nusinersen: antisense oligonucleotide to increase SMN protein production in spinal muscular atrophy. , 2017, Drugs of today.
[60] Hugo M. Botelho,et al. Correction of a Cystic Fibrosis Splicing Mutation by Antisense Oligonucleotides , 2016, Human mutation.
[61] Christophe Béroud,et al. Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy , 2007, Human mutation.
[62] N. Bresolin,et al. Antisense Oligonucleotide Therapy for the Treatment of C9ORF72 ALS/FTD Diseases , 2014, Molecular Neurobiology.
[63] N. Schork,et al. The n-of-1 clinical trial: the ultimate strategy for individualizing medicine? , 2011, Personalized medicine.
[64] E. Androphy,et al. Splicing of a Critical Exon of Human Survival Motor Neuron Is Regulated by a Unique Silencer Element Located in the Last Intron , 2006, Molecular and Cellular Biology.
[65] Helen Donis-Keller,et al. Site specific enzymatic cleavage of RNA , 1979, Nucleic Acids Res..
[66] S. Crooke. RNA directed therapeutics: mechanisms and status , 2013 .
[67] Atul Gupta,et al. Genetic disorders of surfactant protein dysfunction: when to consider and how to investigate , 2016, Archives of Disease in Childhood.
[68] 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.
[69] Paloma H. Giangrande,et al. Current progress on aptamer-targeted oligonucleotide therapeutics. , 2013, Therapeutic delivery.
[70] M. Yeadon,et al. Uptake, efficacy, and systemic distribution of naked, inhaled short interfering RNA (siRNA) and locked nucleic acid (LNA) antisense. , 2011, Molecular therapy : the journal of the American Society of Gene Therapy.
[71] K. Nagaraju,et al. Potential of oligonucleotide-mediated exon-skipping therapy for Duchenne muscular dystrophy , 2007, Expert opinion on biological therapy.
[72] C. Férec,et al. Assessing the Disease-Liability of Mutations in CFTR. , 2012, Cold Spring Harbor perspectives in medicine.
[73] Shuhui Lim,et al. ANTISENSE OLIGONUCLEOTIDES: FROM DESIGN TO THERAPEUTIC APPLICATION , 2006, Clinical and experimental pharmacology & physiology.
[74] R. Juliano,et al. The delivery of therapeutic oligonucleotides , 2016, Nucleic acids research.
[75] A. Hamvas. Evaluation and management of inherited disorders of surfactant metabolism. , 2010, Chinese medical journal.
[76] M. Knowles,et al. Correction of Aberrant Splicing of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene by Antisense Oligonucleotides* , 1999, The Journal of Biological Chemistry.
[77] A. Bush,et al. Corticosteroids in respiratory diseases in children. , 2012, American journal of respiratory and critical care medicine.
[78] J. Kreindler. Cystic fibrosis: exploiting its genetic basis in the hunt for new therapies. , 2010, Pharmacology & therapeutics.
[79] C. Bennett,et al. RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. , 2010, Annual review of pharmacology and toxicology.
[80] R Kole,et al. Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients. , 2000, Proceedings of the National Academy of Sciences of the United States of America.
[81] J. Whitsett,et al. Diseases of pulmonary surfactant homeostasis. , 2015, Annual review of pathology.
[82] Michael J Gait,et al. A chemical view of oligonucleotides for exon skipping and related drug applications. , 2014, Nucleic acid therapeutics.
[83] A. Burdick,et al. Sequence motifs associated with hepatotoxicity of locked nucleic acid—modified antisense oligonucleotides , 2014, Nucleic acids research.
[84] S. Agrawal,et al. Pharmacokinetics of Antisense Oligonucleotides , 1995, Clinical pharmacokinetics.
[85] A. Hamvas,et al. Genotype-phenotype correlations for infants and children with ABCA3 deficiency. , 2014, American journal of respiratory and critical care medicine.
[86] C. Stein,et al. FDA-Approved Oligonucleotide Therapies in 2017. , 2017, Molecular therapy : the journal of the American Society of Gene Therapy.
[87] Xiaohong Huang,et al. Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. , 2015, The New England journal of medicine.
[88] J. Karras,et al. In Vitro and In Vivo Inhibition of Interleukin (IL)-5–Mediated Eosinopoiesis by Murine IL-5R α Antisense Oligonucleotide , 2001 .
[89] Stanley T Crooke,et al. Cellular uptake and trafficking of antisense oligonucleotides , 2017, Nature Biotechnology.
[90] anastasia. khvorova,et al. The chemical evolution of oligonucleotide therapies of clinical utility , 2017, Nature Biotechnology.
[91] M. Lindsay,et al. Clinical potential of oligonucleotide-based therapeutics in the respiratory system. , 2017, Pharmacology & therapeutics.
[92] I. Lerer,et al. Mutation spectrum in Jewish cystic fibrosis patients in Israel: Implication to carrier screening , 2005, American journal of medical genetics. Part A.