Annemieke Aartsma-Rus

Publications

• Development of a Web Course on Gene Therapy by the International Consortium of Gene Therapy
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Mar, 2014  |   Pubmed ID: 24584076
• The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in Mdx Mice
  Molecular Therapy. Nucleic Acids. 2014  |   Pubmed ID: 24549299
• Antisense-mediated Exon Skipping: Networking to Meet Opportunities and to Overcome Challenges
  Nucleic Acid Therapeutics. Feb, 2014  |   Pubmed ID: 24506778
• Antisense-mediated Exon Skipping: Taking Advantage of a Trick from Mother Nature to Treat Rare Genetic Diseases
  Experimental Cell Research. Jan, 2014  |   Pubmed ID: 24486759
• A Novel Feed-Forward Loop Between ARIH2 E3-Ligase and PABPN1 Regulates Aging-Associated Muscle Degeneration
  The American Journal of Pathology. Apr, 2014  |   Pubmed ID: 24486325
• Low Dystrophin Levels in Heart Can Delay Heart Failure in Mdx Mice
  Journal of Molecular and Cellular Cardiology. Apr, 2014  |   Pubmed ID: 24486194
• Fibronectin is a Serum Biomarker for Duchenne Muscular Dystrophy
  Proteomics. Clinical Applications. Jan, 2014  |   Pubmed ID: 24458521
• Novel Ex Vivo Culture Method for the Study of Dupuytren's Disease: Effects of TGFβ Type 1 Receptor Modulation by Antisense Oligonucleotides
  Molecular Therapy. Nucleic Acids. 2014  |   Pubmed ID: 24448195
• Preventing Formation of Toxic N-terminal Huntingtin Fragments Through Antisense Oligonucleotide-mediated Protein Modification
  Nucleic Acid Therapeutics. Feb, 2014  |   Pubmed ID: 24380395
• Peptide Conjugation of 2'-O-methyl Phosphorothioate Antisense Oligonucleotides Enhances Cardiac Uptake and Exon Skipping in Mdx Mice
  Nucleic Acid Therapeutics. Feb, 2014  |   Pubmed ID: 24320790
• A 3-base Pair Deletion, C.9711_9713del, in DMD Results in Intellectual Disability Without Muscular Dystrophy
  European Journal of Human Genetics : EJHG. Apr, 2014  |   Pubmed ID: 23900271
• Autophagy is Impaired in the Tibialis Anterior of Dystrophin Null Mice
  PLoS Currents. 2013  |   Pubmed ID: 24292657
• Generation of Embryonic Stem Cells and Mice for Duchenne Research
  PLoS Currents. 2013  |   Pubmed ID: 24057032
• DMD Transcript Imbalance Determines Dystrophin Levels
  FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology. Dec, 2013  |   Pubmed ID: 23975932
• 194th ENMC International Workshop. 3rd ENMC Workshop on Exon Skipping: Towards Clinical Application of Antisense-mediated Exon Skipping for Duchenne Muscular Dystrophy 8-10 December 2012, Naarden, The Netherlands
  Neuromuscular Disorders : NMD. Nov, 2013  |   Pubmed ID: 23890933
• Antisense-oligonucleotide Mediated Exon Skipping in Activin-receptor-like Kinase 2: Inhibiting the Receptor That is Overactive in Fibrodysplasia Ossificans Progressiva
  PloS One. 2013  |   Pubmed ID: 23861958
• Exon Skipping and Gene Transfer Restore Dystrophin Expression in Human Induced Pluripotent Stem Cells-cardiomyocytes Harboring DMD Mutations
  Stem Cells and Development. Oct, 2013  |   Pubmed ID: 23829870
• Exon Skipping and Gene Transfer Restore Dystrophin Expression in HiPSC-cardiomyocytes Harbouring DMD Mutations
  Stem Cells and Development. Jun, 2013  |   Pubmed ID: 23786351
• Dystrophin-deficient Pigs Provide New Insights into the Hierarchy of Physiological Derangements of Dystrophic Muscle
  Human Molecular Genetics. Nov, 2013  |   Pubmed ID: 23784375
• Ataxin-3 Protein Modification As a Treatment Strategy for Spinocerebellar Ataxia Type 3: Removal of the CAG Containing Exon
  Neurobiology of Disease. Oct, 2013  |   Pubmed ID: 23659897
• Dose-dependent Pharmacokinetic Profiles of 2'-O-methyl Phosphorothioate Antisense Oligonucleotidesin Mdx Mice
  Nucleic Acid Therapeutics. Jun, 2013  |   Pubmed ID: 23634945
• Innovating Therapies for Muscle Diseases
  Handbook of Clinical Neurology. 2013  |   Pubmed ID: 23622373
• Low Dystrophin Levels Increase Survival and Improve Muscle Pathology and Function in Dystrophin/utrophin Double-knockout Mice
  FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology. Jun, 2013  |   Pubmed ID: 23460734
• Traduire La Révolution Génomique : La Nécessité D'un Consortium De Thérapie Génique International Pour Les Maladies Monogéniques
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Feb, 2013  |   Pubmed ID: 23369965
• Advances in Therapeutic RNA-targeting
  New Biotechnology. Mar, 2013  |   Pubmed ID: 23369867
• Inhibition of IL-1 Signaling by Antisense Oligonucleotide-mediated Exon Skipping of IL-1 Receptor Accessory Protein (IL-1RAcP)
  Molecular Therapy. Nucleic Acids. 2013  |   Pubmed ID: 23340324
• Guidance in Social and Ethical Issues Related to Clinical, Diagnostic Care and Novel Therapies for Hereditary Neuromuscular Rare Diseases: "translating" the Translational
  PLoS Currents. 2013  |   Pubmed ID: 23330068
• Isoforme Antisens Médiée Par Commutation De Récepteurs Stéroïdes Coactivateur-1 Dans Le Noyau Central De L'amygdale Du Cerveau De Souris
  BMC Neuroscience. 2013  |   Pubmed ID: 23294837
• Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models
  Molecular Therapy. Nucleic Acids. 2012  |   Pubmed ID: 23344236
• The Effect of 6-thioguanine on Alternative Splicing and Antisense-mediated Exon Skipping Treatment for Duchenne Muscular Dystrophy
  PLoS Currents. 2012  |   Pubmed ID: 23259153
• Digenic Inheritance of an SMCHD1 Mutation and an FSHD-permissive D4Z4 Allele Causes Facioscapulohumeral Muscular Dystrophy Type 2
  Nature Genetics. Dec, 2012  |   Pubmed ID: 23143600
• Gene Therapy for Duchenne Muscular Dystrophy
  Current Opinion in Neurology. Oct, 2012  |   Pubmed ID: 22892952
• Antisense Oligonucleotide Mediated Exon Skipping As a Potential Strategy for the Treatment of a Variety of Inflammatory Diseases Such As Rheumatoid Arthritis
  Annals of the Rheumatic Diseases. Apr, 2012  |   Pubmed ID: 22460143
• Overview on AON Design
  Methods in Molecular Biology (Clifton, N.J.). 2012  |   Pubmed ID: 22454058
• Overview on DMD Exon Skipping
  Methods in Molecular Biology (Clifton, N.J.). 2012  |   Pubmed ID: 22454057
• Overview on Applications of Antisense-mediated Exon Skipping
  Methods in Molecular Biology (Clifton, N.J.). 2012  |   Pubmed ID: 22454056
• Splice Modulating Therapies for Human Disease
  Cell. Mar, 2012  |   Pubmed ID: 22424220
• The Effects of Low Levels of Dystrophin on Mouse Muscle Function and Pathology
  PloS One. 2012  |   Pubmed ID: 22359642
• Comparison of Skeletal Muscle Pathology and Motor Function of Dystrophin and Utrophin Deficient Mouse Strains
  Neuromuscular Disorders : NMD. May, 2012  |   Pubmed ID: 22284942
• Assessment of Cardiac Function in Three Mouse Dystrophinopathies by Magnetic Resonance Imaging
  Neuromuscular Disorders : NMD. May, 2012  |   Pubmed ID: 22209498
• Phage Display Screening Without Repetitious Selection Rounds
  Analytical Biochemistry. Feb, 2012  |   Pubmed ID: 22178910
• Prednisolone Treatment Does Not Interfere with 2'-O-methyl Phosphorothioate Antisense-mediated Exon Skipping in Duchenne Muscular Dystrophy
  Human Gene Therapy. Mar, 2012  |   Pubmed ID: 22017442
• Réglementation Spécifique De Type Cellulaire De La Signalisation De La Myostatine
  The FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology. Dec, 2011  |   Pubmed ID: 22202673
• Dystrophin Quantification and Clinical Correlations in Becker Muscular Dystrophy: Implications for Clinical Trials
  Brain : a Journal of Neurology. Dec, 2011  |   Pubmed ID: 22102647
• Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide
  PloS One. 2011  |   Pubmed ID: 21909428
• Antisense-mediated RNA Targeting: Versatile and Expedient Genetic Manipulation in the Brain
  Frontiers in Molecular Neuroscience. 2011  |   Pubmed ID: 21811437
• Opportunities and Challenges for the Development of Antisense Treatment in Neuromuscular Disorders
  Expert Opinion on Biological Therapy. Aug, 2011  |   Pubmed ID: 21510827
• Exon Double Sauter Dans La Myostatine Et Dystrophine Pour La Dystrophie Musculaire De Duchenne
  BMC Medical Genomics. 2011  |   Pubmed ID: 21507246
• Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD
  Molecular Therapy : the Journal of the American Society of Gene Therapy. May, 2011  |   Pubmed ID: 21468001
• Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy
  The New England Journal of Medicine. Apr, 2011  |   Pubmed ID: 21428760
• The Risks of Therapeutic Misconception and Individual Patient (n=1) "trials" in Rare Diseases Such As Duchenne Dystrophy
  Neuromuscular Disorders : NMD. Jan, 2011  |   Pubmed ID: 21051233
• Antagonistes De BMP Améliorer La Différenciation Myogénique Et D'améliorer Le Phénotype Dystrophique Dans Un Modèle De Souris DMD
  Neurobiology of Disease. Feb, 2011  |   Pubmed ID: 20940052
• New Insights in Gene-derived Therapy: the Example of Duchenne Muscular Dystrophy
  Annals of the New York Academy of Sciences. Dec, 2010  |   Pubmed ID: 21121926
• Antisense-mediated Modulation of Splicing: Therapeutic Implications for Duchenne Muscular Dystrophy
  RNA Biology. Jul-Aug, 2010  |   Pubmed ID: 20523110
• Accurate Quantification of Dystrophin MRNA and Exon Skipping Levels in Duchenne Muscular Dystrophy
  Laboratory Investigation; a Journal of Technical Methods and Pathology. Sep, 2010  |   Pubmed ID: 20458276
• Preclinical PK and PD Studies on 2'-O-methyl-phosphorothioate RNA Antisense Oligonucleotides in the Mdx Mouse Model
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Jun, 2010  |   Pubmed ID: 20407428
• Exonic Sequences Provide Better Targets for Antisense Oligonucleotides Than Splice Site Sequences in the Modulation of Duchenne Muscular Dystrophy Splicing
  Oligonucleotides. Apr, 2010  |   Pubmed ID: 20377429
• A 3 Months Mild Functional Test Regime Does Not Affect Disease Parameters in Young Mdx Mice
  Neuromuscular Disorders : NMD. Apr, 2010  |   Pubmed ID: 20307983
• Therapeutic Exon Skipping for Dysferlinopathies?
  European Journal of Human Genetics : EJHG. Aug, 2010  |   Pubmed ID: 20145676
• Comparative Analysis of Antisense Oligonucleotide Sequences Targeting Exon 53 of the Human DMD Gene: Implications for Future Clinical Trials
  Neuromuscular Disorders : NMD. Feb, 2010  |   Pubmed ID: 20079639
• Progress in Therapeutic Antisense Applications for Neuromuscular Disorders
  European Journal of Human Genetics : EJHG. Feb, 2010  |   Pubmed ID: 19809477
• Development of Antisense-mediated Exon Skipping As a Treatment for Duchenne Muscular Dystrophy
  Annals of the New York Academy of Sciences. Sep, 2009  |   Pubmed ID: 19796079
• Less is More: Therapeutic Exon Skipping for Duchenne Muscular Dystrophy
  Lancet Neurology. Oct, 2009  |   Pubmed ID: 19713153
• [Experimental Therapy in Duchenne Muscular Dystrophy]
  Nederlands Tijdschrift Voor Geneeskunde. May, 2009  |   Pubmed ID: 19475867
• Antisense-mediated Exon Skipping to Correct IL-12Rbeta1 Deficiency in T Cells
  Blood. May, 2009  |   Pubmed ID: 19258592
• Theoretic Applicability of Antisense-mediated Exon Skipping for Duchenne Muscular Dystrophy Mutations
  Human Mutation. Mar, 2009  |   Pubmed ID: 19156838
• In Vivo Comparison of 2'-O-methyl Phosphorothioate and Morpholino Antisense Oligonucleotides for Duchenne Muscular Dystrophy Exon Skipping
  The Journal of Gene Medicine. Mar, 2009  |   Pubmed ID: 19140108
• Guidelines for Antisense Oligonucleotide Design and Insight into Splice-modulating Mechanisms
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Mar, 2009  |   Pubmed ID: 18813282
• Assessment of the Feasibility of Exon 45-55 Multiexon Skipping for Duchenne Muscular Dystrophy
  BMC Medical Genetics. 2008  |   Pubmed ID: 19046429
• The Therapeutic Potential of Antisense-mediated Exon Skipping
  Current Opinion in Molecular Therapeutics. Apr, 2008  |   Pubmed ID: 18386226
• Local Dystrophin Restoration with Antisense Oligonucleotide PRO051
  The New England Journal of Medicine. Dec, 2007  |   Pubmed ID: 18160687
• Antisense-mediated Exon Skipping: a Versatile Tool with Therapeutic and Research Applications
  RNA (New York, N.Y.). Oct, 2007  |   Pubmed ID: 17684229
• Antisense-induced Exon Skipping for Duplications in Duchenne Muscular Dystrophy
  BMC Medical Genetics. 2007  |   Pubmed ID: 17612397
• Entries in the Leiden Duchenne Muscular Dystrophy Mutation Database: an Overview of Mutation Types and Paradoxical Cases That Confirm the Reading-frame Rule
  Muscle & Nerve. Aug, 2006  |   Pubmed ID: 16770791
• Exploring the Frontiers of Therapeutic Exon Skipping for Duchenne Muscular Dystrophy by Double Targeting Within One or Multiple Exons
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Sep, 2006  |   Pubmed ID: 16753346
• Gene Expression Profiling to Monitor Therapeutic and Adverse Effects of Antisense Therapies for Duchenne Muscular Dystrophy
  Pharmacogenomics. Apr, 2006  |   Pubmed ID: 16610940
• Functional Analysis of 114 Exon-internal AONs for Targeted DMD Exon Skipping: Indication for Steric Hindrance of SR Protein Binding Sites
  Oligonucleotides. Dec, 2005  |   Pubmed ID: 16396622
• Targeted Exon Skipping in Transgenic HDMD Mice: A Model for Direct Preclinical Screening of Human-specific Antisense Oligonucleotides
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Aug, 2004  |   Pubmed ID: 15294170
• Antisense-induced Multiexon Skipping for Duchenne Muscular Dystrophy Makes More Sense
  American Journal of Human Genetics. Jan, 2004  |   Pubmed ID: 14681829
• Therapeutic Antisense-induced Exon Skipping in Cultured Muscle Cells from Six Different DMD Patients
  Human Molecular Genetics. Apr, 2003  |   Pubmed ID: 12668614
• Targeted Exon Skipping As a Potential Gene Correction Therapy for Duchenne Muscular Dystrophy
  Neuromuscular Disorders : NMD. Oct, 2002  |   Pubmed ID: 12206800