标题
Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases
作者
关键词
-
出版物
Journal of Neuromuscular Diseases
Volume -, Issue -, Pages 1-16
出版商
IOS Press
发表日期
2021-06-05
DOI
10.3233/jnd-200560
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Inclisiran: First Approval
- (2021) Yvette N. Lamb DRUGS
- Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides
- (2021) Yuichiro Tone et al. Nucleic Acid Therapeutics
- Delivery of oligonucleotide‐based therapeutics: challenges and opportunities
- (2021) Suzan M Hammond et al. EMBO Molecular Medicine
- Golodirsen: First Approval
- (2020) Young-A Heo DRUGS
- Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
- (2020) Diane E. Frank et al. NEUROLOGY
- RNA therapeutics on the rise
- (2020) Feng Wang et al. NATURE REVIEWS DRUG DISCOVERY
- Advances in oligonucleotide drug delivery
- (2020) Thomas C. Roberts et al. NATURE REVIEWS DRUG DISCOVERY
- Trends in incidence, prevalence, and mortality of neuromuscular disease in Ontario, Canada: A population-based retrospective cohort study (2003-2014)
- (2019) Louise Rose et al. PLoS One
- Targeting Huntingtin Expression in Patients with Huntington’s Disease
- (2019) Sarah J. Tabrizi et al. NEW ENGLAND JOURNAL OF MEDICINE
- Autoimmune response and its long‐term consequences after exon‐skipping therapy in a Duchenne Muscular Dystrophy mouse model
- (2019) Joel Z. Nordin et al. JOURNAL OF PATHOLOGY
- Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases
- (2019) Maria K. Tsoumpra et al. EBioMedicine
- The use of genetically humanized animal models for personalized medicine approaches
- (2019) Annemieke Aartsma-Rus et al. Disease Models & Mechanisms
- Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice
- (2019) Arnaud F. Klein et al. JOURNAL OF CLINICAL INVESTIGATION
- A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system
- (2019) Julia F. Alterman et al. NATURE BIOTECHNOLOGY
- Wrangling RNA: Antisense oligonucleotides for neurological disorders
- (2019) Kevin Talbot et al. Science Translational Medicine
- Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
- (2019) Jinkuk Kim et al. NEW ENGLAND JOURNAL OF MEDICINE
- News Feature: Gene therapy successes point to better therapies
- (2019) Elie Dolgin PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs
- (2019) Akin Akinc et al. Nature Nanotechnology
- Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
- (2018) Eugenio Mercuri et al. NEW ENGLAND JOURNAL OF MEDICINE
- RNA-mediated therapies in myotonic dystrophy
- (2018) Sarah J. Overby et al. DRUG DISCOVERY TODAY
- Patisiran: First Global Approval
- (2018) Sheridan M. Hoy DRUGS
- Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)
- (2018) Hasane Ratni et al. JOURNAL OF MEDICINAL CHEMISTRY
- Extensive and Prolonged Restoration of Dystrophin Expression with Vivo-Morpholino-Mediated Multiple Exon Skipping in Dystrophic Dogs
- (2018) Toshifumi Yokota et al. Nucleic Acid Therapeutics
- Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment
- (2018) Michael J. Gait et al. Nucleic Acid Therapeutics
- Antisense oligonucleotides: the next frontier for treatment of neurological disorders
- (2017) Carlo Rinaldi et al. Nature Reviews Neurology
- Polyglutamine spinocerebellar ataxias — from genes to potential treatments
- (2017) Henry L. Paulson et al. NATURE REVIEWS NEUROSCIENCE
- Prevalence, incidence and carrier frequency of 5q–linked spinal muscular atrophy – a literature review
- (2017) Ingrid E. C. Verhaart et al. Orphanet Journal of Rare Diseases
- FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga
- (2017) Annemieke Aartsma-Rus et al. Nucleic Acid Therapeutics
- Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice
- (2017) Lodewijk J.A. Toonen et al. Molecular Therapy-Nucleic Acids
- Duchenne Muscular Dystrophy Myogenic Cells from Urine-Derived Stem Cells Recapitulate the Dystrophin Genotype and Phenotype
- (2016) Maria Sofia Falzarano et al. HUMAN GENE THERAPY
- Welcome to the splice age: antisense oligonucleotide–mediated exon skipping gains wider applicability
- (2016) Elizabeth M. McNally et al. JOURNAL OF CLINICAL INVESTIGATION
- Developmental milestones in type I spinal muscular atrophy
- (2016) Roberto De Sanctis et al. NEUROMUSCULAR DISORDERS
- Molecular characterization and copy number of SMN1, SMN2 and NAIP in Chinese patients with spinal muscular atrophy and unrelated healthy controls
- (2015) Ping Fang et al. BMC MUSCULOSKELETAL DISORDERS
- Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy
- (2015) M. Gramlich et al. EMBO Molecular Medicine
- Ameliorating pathogenesis by removing an exon containing a missense mutation: a potential exon-skipping therapy for laminopathies
- (2015) J Scharner et al. GENE THERAPY
- Correction of a Cystic Fibrosis Splicing Mutation by Antisense Oligonucleotides
- (2015) Susana Igreja et al. HUMAN MUTATION
- The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations
- (2015) Catherine L. Bladen et al. HUMAN MUTATION
- Reengineering a transmembrane protein to treat muscular dystrophy using exon skipping
- (2015) Quan Q. Gao et al. JOURNAL OF CLINICAL INVESTIGATION
- Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease
- (2014) Nicholas P Clayton et al. Molecular Therapy-Nucleic Acids
- Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy
- (2013) Melissa S. Cobb et al. HUMAN MOLECULAR GENETICS
- Novel Hydrophobically Modified Asymmetric RNAi Compounds (sd-rxRNA) Demonstrate Robust Efficacy in the Eye
- (2013) Michael Byrne et al. JOURNAL OF OCULAR PHARMACOLOGY AND THERAPEUTICS
- A Chemical View of Oligonucleotides for Exon Skipping and Related Drug Applications
- (2013) Peter Järver et al. Nucleic Acid Therapeutics
- Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression
- (2012) Dongbo Yu et al. CELL
- Single-Stranded siRNAs Activate RNAi in Animals
- (2012) Walt F. Lima et al. CELL
- The incidence and prevalence of Huntington's disease: A systematic review and meta-analysis
- (2012) Tamara Pringsheim et al. MOVEMENT DISORDERS
- Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery
- (2012) Y. Aoki et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Renal uptake and tolerability of a 2′-O-methoxyethyl modified antisense oligonucleotide (ISIS 113715) in monkey
- (2012) Scott P. Henry et al. TOXICOLOGY
- 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
- (2012) Alberto Malerba et al. Molecular Therapy-Nucleic Acids
- Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment
- (2012) Corinne Betts et al. Molecular Therapy-Nucleic Acids
- In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse
- (2010) Yoshitsugu Aoki et al. MOLECULAR THERAPY
- Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs
- (2009) Toshifumi Yokota et al. ANNALS OF NEUROLOGY
- Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management
- (2009) Katharine Bushby et al. LANCET NEUROLOGY
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