- Home
- Publications
- Publication Search
- Publication Details
Title
Translational development of splice-modifying antisense oligomers
Authors
Keywords
-
Journal
EXPERT OPINION ON BIOLOGICAL THERAPY
Volume 17, Issue 1, Pages 15-30
Publisher
Informa UK Limited
Online
2016-11-02
DOI
10.1080/14712598.2017.1250880
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
- (2016) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Contributions of Japanese patients to development of antisense therapy for DMD
- (2016) Masafumi Matsuo et al. BRAIN & DEVELOPMENT
- Pharmacokinetics, pharmacodynamics, and efficacy of a small-moleculeSMN2splicing modifier in mouse models of spinal muscular atrophy
- (2016) Xin Zhao et al. HUMAN MOLECULAR GENETICS
- Antisense molecules: A new class of drugs
- (2016) Daniel P. Potaczek et al. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
- Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy
- (2016) Volker Straub et al. LANCET NEUROLOGY
- The case for eteplirsen: Paving the way for precision medicine
- (2016) M. Carrie Miceli et al. MOLECULAR GENETICS AND METABOLISM
- DMDgenotypes and loss of ambulation in the CINRG Duchenne Natural History Study
- (2016) Luca Bello et al. NEUROLOGY
- Categorizing natural history trajectories of ambulatory function measured by the 6-minute walk distance in patients with Duchenne muscular dystrophy
- (2016) Eugenio Mercuri et al. NEUROMUSCULAR DISORDERS
- Revised North Star Ambulatory Assessment for Young Boys with Duchenne Muscular Dystrophy
- (2016) Eugenio Mercuri et al. PLoS One
- Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy
- (2016) Suzan M. Hammond et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice
- (2016) Gang Han et al. Nature Communications
- Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies
- (2016) Luca Bello et al. Scientific Reports
- Fructose Promotes Uptake and Activity of Oligonucleotides With Different Chemistries in a Context-dependent Manner in mdx Mice
- (2016) Limin Cao et al. Molecular Therapy-Nucleic Acids
- Mechanisms and Regulation of Alternative Pre-mRNA Splicing
- (2015) Yeon Lee et al. Annual Review of Biochemistry
- Improving clinical trial design for Duchenne muscular dystrophy
- (2015) Luciano Merlini et al. BMC Neurology
- Antisense oligonucleotide drugs for Duchenne muscular dystrophy: how far have we come and what does the future hold?
- (2015) Ashley Guncay et al. Future Medicinal Chemistry
- Oligonucleotide therapeutics: chemistry, delivery and clinical progress
- (2015) Vivek K Sharma et al. Future Medicinal Chemistry
- The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations
- (2015) Catherine L. Bladen et al. HUMAN MUTATION
- The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials
- (2015) Valeria Ricotti et al. JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY
- Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
- (2015) Kathrin Meyer et al. MOLECULAR THERAPY
- SMN2 splice modulators enhance U1–pre-mRNA association and rescue SMA mice
- (2015) James Palacino et al. Nature Chemical Biology
- Exon 53 skipping of the dystrophin gene in patients with Duchenne muscular dystrophy by systemic administration of NS-065/NCNP-01: A phase 1, dose escalation, first-in-human study
- (2015) H. Komaki et al. NEUROMUSCULAR DISORDERS
- Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy
- (2015) Stephen Lynn et al. NEUROMUSCULAR DISORDERS
- 2′-Fluoro-modified phosphorothioate oligonucleotide can cause rapid degradation of P54nrb and PSF
- (2015) Wen Shen et al. NUCLEIC ACIDS RESEARCH
- Plastin 3 Expression Does Not Modify Spinal Muscular Atrophy Severity in the ∆7 SMA Mouse
- (2015) Vicki L. McGovern et al. PLoS One
- The emperor's new dystrophin: finding sense in the noise
- (2015) S.D. Wilton et al. TRENDS IN MOLECULAR MEDICINE
- Antisense oligonucleotide development for the treatment of muscular dystrophies
- (2015) Tri Le Bao et al. Expert Opinion on Orphan Drugs
- Evaluation of 2’-Deoxy-2’-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy
- (2015) Silvana M G Jirka et al. Molecular Therapy-Nucleic Acids
- Pseudoexon activation increases phenotype severity in a Becker muscular dystrophy patient
- (2015) Kane Greer et al. Molecular Genetics & Genomic Medicine
- New developments in exon skipping and splice modulation therapies for neuromuscular diseases
- (2014) Aleksander Touznik et al. EXPERT OPINION ON BIOLOGICAL THERAPY
- Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models
- (2014) Erkan Y. Osman et al. HUMAN MOLECULAR GENETICS
- Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study
- (2014) Thomas Voit et al. LANCET NEUROLOGY
- Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice
- (2014) Nicolas Wein et al. NATURE MEDICINE
- Biomarkers and surrogate endpoints in Duchenne: Meeting report
- (2014) Annemieke Aartsma-Rus et al. NEUROMUSCULAR DISORDERS
- Dystrophin as a therapeutic biomarker: Are we ignoring data from the past?
- (2014) Steve D. Wilton et al. NEUROMUSCULAR DISORDERS
- Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes
- (2014) Marika Pane et al. PLoS One
- SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
- (2014) N. A. Naryshkin et al. SCIENCE
- Exon-Skipping Therapy: A Roadblock, Detour, or Bump in the Road?
- (2014) E. P. Hoffman et al. Science Translational Medicine
- Exon identity crisis: disease-causing mutations that disrupt the splicing code
- (2014) Timothy Sterne-Weiler et al. GENOME BIOLOGY
- What Can We Learn From Clinical Trials of Exon Skipping for DMD?
- (2014) Qi-long Lu et al. Molecular Therapy-Nucleic Acids
- Eteplirsen for the treatment of Duchenne muscular dystrophy
- (2013) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Spinal muscular atrophy: An update on therapeutic progress
- (2013) Joonbae Seo et al. BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE
- Strategies for Postmarketing Surveillance of Drugs for Rare Diseases
- (2013) A S Kesselheim et al. CLINICAL PHARMACOLOGY & THERAPEUTICS
- How genetic modifiers influence the phenotype of spinal muscular atrophy and suggest future therapeutic approaches
- (2013) Brunhilde Wirth et al. CURRENT OPINION IN GENETICS & DEVELOPMENT
- Detecting meaningful change using the North Star Ambulatory Assessment in Duchenne muscular dystrophy
- (2013) Anna G Mayhew et al. DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY
- Pluronic–PEI copolymers enhance exon-skipping of 2′-O-methyl phosphorothioate oligonucleotide in cell culture and dystrophic mdx mice
- (2013) M Wang et al. GENE THERAPY
- A Novel Morpholino Oligomer Targeting ISS-N1 Improves Rescue of Severe Spinal Muscular Atrophy Transgenic Mice
- (2013) Haiyan Zhou et al. HUMAN GENE THERAPY
- Clinical characterisation of Becker muscular dystrophy patients predicts favourable outcome in exon-skipping therapy
- (2013) J. C. van den Bergen et al. JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY
- An intronic structure enabled by a long-distance interaction serves as a novel target for splicing correction in spinal muscular atrophy
- (2013) Natalia N. Singh et al. NUCLEIC ACIDS RESEARCH
- Species-specific Inflammatory Responses as a Primary Component for the Development of Glomerular Lesions in Mice and Monkeys Following Chronic Administration of a Second-generation Antisense Oligonucleotide
- (2013) Kendall S. Frazier et al. TOXICOLOGIC PATHOLOGY
- Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency
- (2013) HaiFang Yin et al. Molecular Therapy-Nucleic Acids
- LTBP4genotype predicts age of ambulatory loss in duchenne muscular dystrophy
- (2012) Kevin M. Flanigan et al. ANNALS OF NEUROLOGY
- Evaluation of SMN Protein, Transcript, and Copy Number in the Biomarkers for Spinal Muscular Atrophy (BforSMA) Clinical Study
- (2012) Thomas O. Crawford et al. PLoS One
- Dantrolene Enhances Antisense-Mediated Exon Skipping in Human and Mouse Models of Duchenne Muscular Dystrophy
- (2012) G. C. Kendall et al. Science Translational Medicine
- Innovative study design for paediatric clinical trials
- (2011) Paola Baiardi et al. EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY
- A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse
- (2011) Paul N. Porensky et al. HUMAN MOLECULAR GENETICS
- A framework for applying unfamiliar trial designs in studies of rare diseases
- (2011) Samir Gupta et al. JOURNAL OF CLINICAL EPIDEMIOLOGY
- Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
- (2011) Sebahattin Cirak et al. LANCET
- Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model
- (2011) Yimin Hua et al. NATURE
- Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy
- (2011) Nathalie M. Goemans et al. NEW ENGLAND JOURNAL OF MEDICINE
- The FSHD Atrophic Myotube Phenotype Is Caused by DUX4 Expression
- (2011) Céline Vanderplanck et al. PLoS One
- Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
- (2011) M. A. Passini et al. Science Translational Medicine
- RNA-targeted splice-correction therapy for neuromuscular disease
- (2010) M. J. A. Wood et al. BRAIN
- Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene
- (2010) Kevin M. Flanigan et al. HUMAN MUTATION
- Dystrophin Isoform Induction In Vivo by Antisense-mediated Alternative Splicing
- (2010) Sue Fletcher et al. MOLECULAR THERAPY
- A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease
- (2010) Ans T. van der Ploeg et al. NEW ENGLAND JOURNAL OF MEDICINE
- Identification of a novel muscle targeting peptide in mdx mice
- (2010) Yiqi Seow et al. PEPTIDES
- Antisense-mediated modulation of splicing: Therapeutic implications for Duchenne muscular dystrophy
- (2010) Annemieke Aartsma-Rus RNA Biology
- Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs
- (2009) Toshifumi Yokota et al. ANNALS OF NEUROLOGY
- Development of Antisense-Mediated Exon Skipping as a Treatment for Duchenne Muscular Dystrophy
- (2009) Hans Heemskerk et al. Annals of the New York Academy of Sciences
- Cellular Uptake of Neutral Phosphorodiamidate Morpholino Oligomers
- (2009) Patrick Iversen et al. CURRENT PHARMACEUTICAL BIOTECHNOLOGY
- Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino
- (2009) B Wu et al. GENE THERAPY
- In vivocomparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping
- (2009) Hans A. Heemskerk et al. JOURNAL OF GENE MEDICINE
- 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) Maria Kinali et al. LANCET NEUROLOGY
- Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management
- (2009) Katharine Bushby et al. LANCET NEUROLOGY
- Rational Design of Antisense Oligomers to Induce Dystrophin Exon Skipping
- (2009) Chalermchai Mitrpant et al. MOLECULAR THERAPY
- Cationic PMMA Nanoparticles Bind and Deliver Antisense Oligoribonucleotides Allowing Restoration of Dystrophin Expression in the mdx Mouse
- (2009) Paola Rimessi et al. MOLECULAR THERAPY
- Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice
- (2008) Yimin Hua et al. AMERICAN JOURNAL OF HUMAN GENETICS
- Spinal muscular atrophy
- (2008) Mitchell R Lunn et al. LANCET
- Regulation of SMN Protein Stability
- (2008) B. G. Burnett et al. MOLECULAR AND CELLULAR BIOLOGY
- Improved cell-penetrating peptide–PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle
- (2008) Gabriela D. Ivanova et al. NUCLEIC ACIDS RESEARCH
- Plastin 3 Is a Protective Modifier of Autosomal Recessive Spinal Muscular Atrophy
- (2008) G. E. Oprea et al. SCIENCE
- DMDpseudoexon mutations: splicing efficiency, phenotype, and potential therapy
- (2007) Olga L. Gurvich et al. ANNALS OF NEUROLOGY
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationCreate your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create Now