New developments in exon skipping and splice modulation therapies for neuromuscular diseases
Published 2014 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
New developments in exon skipping and splice modulation therapies for neuromuscular diseases
Authors
Keywords
-
Journal
EXPERT OPINION ON BIOLOGICAL THERAPY
Volume 14, Issue 6, Pages 809-819
Publisher
Informa Healthcare
Online
2014-03-12
DOI
10.1517/14712598.2014.896335
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Dysferlin and Animal Models for Dysferlinopathy
- (2015) Kinji Kobayashi et al. Journal of Toxicologic Pathology
- Skipping Multiple Exons of Dystrophin Transcripts Using Cocktail Antisense Oligonucleotides
- (2014) Yusuke Echigoya et al. Nucleic Acid Therapeutics
- Eteplirsen for the treatment of Duchenne muscular dystrophy
- (2013) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Molecular Therapeutic Strategies for Spinal Muscular Atrophies: Current and Future Clinical Trials
- (2013) Chiara Zanetta et al. CLINICAL THERAPEUTICS
- Oral Corticosteroids and Onset of Cardiomyopathy in Duchenne Muscular Dystrophy
- (2013) Brent J. Barber et al. JOURNAL OF PEDIATRICS
- Mutation Types and Aging Differently Affect Revertant Fiber Expansion in Dystrophic Mdx and Mdx52 Mice
- (2013) Yusuke Echigoya et al. PLoS One
- Correlations Between Change Scores of Measures for Muscle Strength and Motor Function in Individuals with Spinal Muscular Atrophy Types 2 and 3
- (2012) Hui-Yi Wang et al. AMERICAN JOURNAL OF PHYSICAL MEDICINE & REHABILITATION
- Splice Modulating Therapies for Human Disease
- (2012) Pietro Spitali et al. CELL
- Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges
- (2012) Virginia Arechavala-Gomeza et al. CURRENT GENE THERAPY
- Modular Dispensability of Dysferlin C2 Domains Reveals Rational Design for Mini-dysferlin Molecules
- (2012) Bilal A. Azakir et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Dysfunction of the neuromuscular junction in spinal muscular atrophy types 2 and 3
- (2012) R. I. Wadman et al. NEUROLOGY
- 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
- Restoring Dystrophin Expression in Duchenne Muscular Dystrophy Muscle
- (2011) Eric P. Hoffman et al. AMERICAN JOURNAL OF PATHOLOGY
- Antisense drug discovery and development
- (2011) Tsuyoshi Yamamoto et al. Future Medicinal Chemistry
- UMD-DYSF, a novel locus specific database for the compilation and interactive analysis of mutations in the dysferlin gene
- (2011) Gaelle Blandin et al. HUMAN MUTATION
- Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy
- (2011) Mariko Taniguchi-Ikeda et al. NATURE
- Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model
- (2011) Yimin Hua et al. NATURE
- Targeting RNA to treat neuromuscular disease
- (2011) Francesco Muntoni et al. NATURE REVIEWS DRUG DISCOVERY
- Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy
- (2011) Nathalie M. Goemans et al. NEW ENGLAND JOURNAL OF MEDICINE
- Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
- (2011) M. A. Passini et al. Science Translational Medicine
- Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
- (2010) Y. Hua et al. GENES & DEVELOPMENT
- Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model
- (2010) Hans Heemskerk et al. MOLECULAR THERAPY
- Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
- (2010) Kevin D Foust et al. NATURE BIOTECHNOLOGY
- Antisense-mediated modulation of splicing: Therapeutic implications for Duchenne muscular dystrophy
- (2010) Annemieke Aartsma-Rus RNA Biology
- A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy
- (2010) M. Krahn et al. Science Translational Medicine
- Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs
- (2009) Toshifumi Yokota et al. ANNALS OF NEUROLOGY
- A Renaissance for Antisense Oligonucleotide Drugs in Neurology
- (2009) Toshifumi Yokota et al. ARCHIVES OF NEUROLOGY
- Characterization of Lipid Binding Specificities of Dysferlin C2 Domains Reveals Novel Interactions with Phosphoinositides†
- (2009) Christian Therrien et al. BIOCHEMISTRY
- Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations
- (2009) Annemieke Aartsma-Rus et al. HUMAN MUTATION
- Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping
- (2009) Nicolas Wein et al. HUMAN MUTATION
- Membrane Repair Defects in Muscular Dystrophy Are Linked to Altered Interaction between MG53, Caveolin-3, and Dysferlin
- (2009) Chuanxi Cai et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Oligonucleotide-Mediated Survival of Motor Neuron Protein Expression in CNS Improves Phenotype in a Mouse Model of Spinal Muscular Atrophy
- (2009) J. H. Williams et al. JOURNAL OF NEUROSCIENCE
- Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care
- (2009) Katharine Bushby et al. LANCET NEUROLOGY
- Biological Barriers to Therapy with Antisense and siRNA Oligonucleotides
- (2009) R. Juliano et al. MOLECULAR PHARMACEUTICS
- Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos
- (2009) Jon Moulton et al. MOLECULES
- Dysferlinopathies
- (2009) JAndoni Urtizberea et al. NEUROLOGY INDIA
- Limb–girdle muscular dystrophies
- (2008) Michela Guglieri et al. CURRENT OPINION IN NEUROLOGY
- Asymptomatic Sporadic Dysferlinopathy Presenting with Elevation of Serum Creatine Kinase. Typical Distribution of Muscle Involvement Shown by MRI but not by CT
- (2008) Satomi Okahashi et al. INTERNAL MEDICINE
- Clinical and molecular overlap between myopathies and inherited connective tissue diseases
- (2008) N.C. Voermans et al. NEUROMUSCULAR DISORDERS
- G.O.5 Partial functionality of a Mini-dysferlin molecule identified in a patient affected with moderately severe primary dysferlinopathy
- (2008) M. Krahn et al. NEUROMUSCULAR DISORDERS
- Late onset in dysferlinopathy widens the clinical spectrum
- (2008) L. Klinge et al. NEUROMUSCULAR DISORDERS
Add your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload NowCreate your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create Now