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Treatability of the KMT2‐Associated Neurodevelopmental Disorders Using Antisense Oligonucleotide‐Based Treatments.
- Published in:
- Human Mutation, 2024, v. 2024, p. 1, doi. 10.1155/2024/9933129
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- Publication type:
- Article
In vivo comparison of 2′- O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping.
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- Journal of Gene Medicine, 2009, v. 11, n. 3, p. 257, doi. 10.1002/jgm.1288
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- Publication type:
- Article
The RNA-binding profile of the splicing factor SRSF6 in immortalized human pancreatic β-cells.
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- Life Science Alliance, 2021, v. 4, n. 3, p. 1, doi. 10.26508/lsa.202000825
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- Publication type:
- Article
Antisense Oligonucleotide-Mediated Downregulation of IGFBPs Enhances IGF-1 Signaling.
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- Journal of Neuromuscular Diseases, 2024, v. 11, n. 2, p. 299, doi. 10.3233/JND-230118
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- Article
The Dilemma of Choice for Duchenne Patients Eligible for Exon 51 Skipping The European Experience.
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- Journal of Neuromuscular Diseases, 2023, v. 10, n. 3, p. 315, doi. 10.3233/JND-221648
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- Publication type:
- Article
On the use of D2.B10-Dmd<sup>mdx</sup>/J (D2.mdx) Versus C57BL/10ScSn-Dmd<sup>mdx</sup>/J (mdx) Mouse Models for Preclinical Studies on Duchenne Muscular Dystrophy: A Cautionary Note from Members of the TREAT-NMD Advisory Committee on Therapeutics.
- Published in:
- Journal of Neuromuscular Diseases, 2023, v. 10, n. 1, p. 155, doi. 10.3233/JND-221547
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- Article
Author's Response to: Rebuttal to: Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy, Verhaart et al. 2020.
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- Journal of Neuromuscular Diseases, 2021, v. 8, n. 5, p. 867, doi. 10.3233/JND-219004
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- Publication type:
- Article
Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy.
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- Journal of Neuromuscular Diseases, 2021, v. 8, n. 5, p. 845, doi. 10.3233/JND-200524
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- Publication type:
- Article
Sharing "Negative" Results in Neuromuscular Research: A Positive Experience.
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- Journal of Neuromuscular Diseases, 2021, v. 8, n. 5, p. 765, doi. 10.3233/JND-219007
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- Publication type:
- Article
Dystrophin deficiency leads to dysfunctional glutamate clearance in iPSC derived astrocytes.
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- Translational Psychiatry, 2019, v. 9, n. 1, p. N.PAG, doi. 10.1038/s41398-019-0535-1
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- Publication type:
- Article
Imperatives for DUCHENNE MD: a Simplified Guide to Comprehensive Care for Duchenne Muscular Dystrophy.
- Published in:
- PLoS Currents, 2015, p. 276, doi. 10.1371/currents.md.87770501e86f36f1c71e0a5882ed9ba1
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- Publication type:
- Article
Generation of Embryonic Stem Cells and Mice for Duchenne Research.
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- PLoS Currents, 2013, p. 264, doi. 10.1371/currents.md.cbf1d33001de80923ce674302cad7925
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- Publication type:
- Article
The Effect of 6-Thioguanine on Alternative Splicing and Antisense-Mediated Exon Skipping Treatment for Duchenne Muscular Dystrophy.
- Published in:
- PLoS Currents, 2012, p. 547, doi. 10.1371/currents.md.597d700f92eaa70de261ea0d91821377
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- Publication type:
- Article
A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.
- Published in:
- PLoS ONE, 2018, v. 13, n. 10, p. 1, doi. 10.1371/journal.pone.0204485
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- Publication type:
- Article
Influence of full-length dystrophin on brain volumes in mouse models of Duchenne muscular dystrophy.
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- PLoS ONE, 2018, v. 13, n. 3, p. 1, doi. 10.1371/journal.pone.0194636
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- Publication type:
- Article
A dystrophic Duchenne mouse model for testing human antisense oligonucleotides.
- Published in:
- PLoS ONE, 2018, v. 13, n. 2, p. 1, doi. 10.1371/journal.pone.0193289
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- Publication type:
- Article
Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy.
- Published in:
- BMC Medical Genomics, 2011, v. 4, n. 1, p. 36, doi. 10.1186/1755-8794-4-36
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- Publication type:
- Article
Natural disease history of the D2-mdx mouse model for Duchenne muscular dystrophy.
- Published in:
- FASEB Journal, 2019, v. 33, n. 7, p. 8110, doi. 10.1096/fj.201802488R
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- Publication type:
- Article
New function of the myostatin/activin type I receptor (ALK4) as a mediator of muscle atrophy and muscle regeneration.
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- FASEB Journal, 2017, v. 31, n. 1, p. 238, doi. 10.1096/fj.201600675r
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- Publication type:
- Article
New function of the myostatin/activin type I receptor (ALK4) as a mediator of muscle atrophy and muscle regeneration.
- Published in:
- FASEB Journal, 2017, v. 31, n. 1, p. 238, doi. 10.1096/fj.201600675R
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- Publication type:
- Article
DMD transcript imbalance determines dystrophin levels.
- Published in:
- FASEB Journal, 2013, v. 27, n. 12, p. 4909, doi. 10.1096/fj.13-232025
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- Publication type:
- Article
Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice.
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- FASEB Journal, 2013, v. 27, n. 6, p. 2484, doi. 10.1096/fj.12-224170
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- Publication type:
- Article
Cell-type specific regulation of myostatin signaling.
- Published in:
- FASEB Journal, 2012, v. 26, n. 4, p. 1462, doi. 10.1096/fj.11-191189
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- Publication type:
- Article
New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.
- Published in:
- Annals of the New York Academy of Sciences, 2010, v. 1214, n. 1, p. 199, doi. 10.1111/j.1749-6632.2010.05836.x
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- Publication type:
- Article
Development of Antisense-Mediated Exon Skipping as a Treatment for Duchenne Muscular Dystrophy.
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- Annals of the New York Academy of Sciences, 2009, v. 1175, n. 1, p. 71, doi. 10.1111/j.1749-6632.2009.04973.x
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- Publication type:
- Article
Development of a Web Course on Gene Therapy by the International Consortium of Gene Therapy.
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- Molecular Therapy, 2014, v. 22, n. 3, p. 482, doi. 10.1038/mt.2014.11
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- Article
Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases.
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- Molecular Therapy, 2013, v. 21, n. 2, p. 266, doi. 10.1038/mt.2013.4
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- Article
RNA-based therapies for genodermatoses.
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- Experimental Dermatology, 2017, v. 26, n. 1, p. 3, doi. 10.1111/exd.13141
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- Article
Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle.
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- Human Molecular Genetics, 2013, v. 22, n. 21, p. 4368, doi. 10.1093/hmg/ddt287
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- Article
The dystrophin gene and cognitive function in the general population.
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- European Journal of Human Genetics, 2015, v. 23, n. 6, p. 837, doi. 10.1038/ejhg.2014.183
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- Article
A 3-base pair deletion, c.9711_9713del, in DMD results in intellectual disability without muscular dystrophy.
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- European Journal of Human Genetics, 2014, v. 22, n. 4, p. 480, doi. 10.1038/ejhg.2013.169
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- Article
Assessment of Behavioral Characteristics With Procedures of Minimal Human Interference in the mdx Mouse Model for Duchenne Muscular Dystrophy.
- Published in:
- Frontiers in Behavioral Neuroscience, 2021, v. 14, p. N.PAG, doi. 10.3389/fnbeh.2020.629043
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- Article
Orthogonal proteomics methods warrant the development of Duchenne muscular dystrophy biomarkers.
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- Clinical Proteomics, 2023, v. 20, n. 1, p. 1, doi. 10.1186/s12014-023-09412-1
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- Article
The IRDiRC Chrysalis Task Force: making rare disease research attractive to companies.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231188979
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- Article
Serum Neurofilament light correlates with CADASIL disease severity and survival.
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- Annals of Clinical & Translational Neurology, 2019, v. 6, n. 1, p. 46, doi. 10.1002/acn3.678
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- Article
The NOTCH3 score: a pre-clinical CADASIL biomarker in a novel human genomic NOTCH3 transgenic mouse model with early progressive vascular NOTCH3 accumulation.
- Published in:
- Acta Neuropathologica Communications, 2015, v. 3, p. 1, doi. 10.1186/s40478-015-0268-1
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- Article
Delivery of oligonucleotide‐based therapeutics: challenges and opportunities.
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- EMBO Molecular Medicine, 2021, v. 13, n. 4, p. 1, doi. 10.15252/emmm.202013243
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- Article
Peripheral blood transcriptome profiling enables monitoring disease progression in dystrophic mice and patients.
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- EMBO Molecular Medicine, 2021, v. 13, n. 4, p. 1, doi. 10.15252/emmm.202013328
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- Publication type:
- Article
Delivery is key: lessons learnt from developing splice-switching antisense therapies.
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- EMBO Molecular Medicine, 2017, v. 9, n. 5, p. 545, doi. 10.15252/emmm.201607199
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- Publication type:
- Article
Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy.
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- EMBO Molecular Medicine, 2015, v. 7, n. 5, p. 562, doi. 10.15252/emmm.201505047
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- Publication type:
- Article
Affinity proteomics within rare diseases: a BIO- NMD study for blood biomarkers of muscular dystrophies.
- Published in:
- EMBO Molecular Medicine, 2014, v. 6, n. 7, p. 918, doi. 10.15252/emmm.201303724
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- Publication type:
- Article
Therapeutic exon skipping for dysferlinopathies?
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- 2010
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- Correction notice
Reply to Lévy et al.
- Published in:
- 2010
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- Publication type:
- Letter
Therapeutic exon skipping for dysferlinopathies?
- Published in:
- European Journal of Human Genetics, 2010, v. 18, n. 8, p. 889, doi. 10.1038/ejhg.2010.4
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- Publication type:
- Article
Progress in therapeutic antisense applications for neuromuscular disorders.
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- European Journal of Human Genetics, 2010, v. 18, n. 2, p. 146, doi. 10.1038/ejhg.2009.160
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- Publication type:
- Article
Digenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2.
- Published in:
- Nature Genetics, 2012, v. 44, n. 12, p. 1370, doi. 10.1038/ng.2454
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- Article
Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 5, p. 830, doi. 10.1038/mt.2011.59
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- Publication type:
- Article
Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 6, p. 1210, doi. 10.1038/mt.2010.72
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- Publication type:
- Article
Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 3, p. 548, doi. 10.1038/mt.2008.205
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- Publication type:
- Article
Exploring the Frontiers of Therapeutic Exon Skipping for Duchenne Muscular Dystrophy by Double Targeting within One or Multiple Exons.
- Published in:
- Molecular Therapy, 2006, v. 14, n. 3, p. 401, doi. 10.1016/j.ymthe.2006.02.022
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- Article