FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga.
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Delivery is key: lessons learnt from developing splice-switching antisense therapiesGenetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy.Towards improved oligonucleotide therapeutics through faster target binding kinetics.Alternative splicing: the pledge, the turn, and the prestige : The key role of alternative splicing in human biological systems.Exon skipping: a first in class strategy for Duchenne muscular dystrophy.2'-O-Methyl RNA/Ethylene-Bridged Nucleic Acid Chimera Antisense Oligonucleotides to Induce Dystrophin Exon 45 Skipping.Modulation of aberrant splicing in human RNA diseases by chemical compounds.RNA splicing and splicing regulator changes in prostate cancer pathologyProgress toward Gene Therapy for Duchenne Muscular Dystrophy.The importance of international collaboration for rare diseases research: a European perspective.Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy.Enhancing the cytotoxicity of chemoradiation with radiation-guided delivery of anti-MGMT morpholino oligonucleotides in non-methylated solid tumors.Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy.Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse ModelSleep Disorders in Childhood Neurogenetic Disorders.Gene therapy for Duchenne muscular dystrophy: balancing good science, marginal efficacy, high emotions and excessive cost.Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues.Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy.Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne.Tween 85-Modified Low Molecular Weight PEI Enhances Exon-Skipping of Antisense Morpholino Oligomer In Vitro and in mdx Mice.Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy.Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy.Effective regeneration of dystrophic muscle using autologous iPSC-derived progenitors with CRISPR-Cas9 mediated precise correction.Myoblots: dystrophin quantification by in-cell western assay for a streamlined development of Duchenne muscular dystrophy (DMD) treatments.Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy.Oligonucleotides Targeting TCF4 Triplet Repeat Expansion Inhibit RNA Foci and Mis-splicing in Fuchs' Dystrophy.Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs.Semi-Automated Analysis of Diaphragmatic Motion with Dynamic Magnetic Resonance Imaging in Healthy Controls and Non-Ambulant Subjects with Duchenne Muscular Dystrophy.Myopenia and precision (P4) medicine.Structure-Activity Relationship Study of Leucyl-3-epi-deoxynegamycin for Potent Premature Termination Codon Readthrough.Mutation-Based Therapy for Duchenne Muscular Dystrophy: Antisense Treatment Arrives in the Clinic.Selection and Identification of Skeletal-Muscle-Targeted RNA Aptamers.Recent Advances in Antisense Oligonucleotide Therapy in Genetic Neuromuscular Diseases.Aptamer-siRNA Chimeras: Discovery, Progress, and Future Prospects.Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.Antibody-Antisense Oligonucleotide Conjugate Downregulates a Key Gene in Glioblastoma Stem Cells.Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers.Analysis of extracellular mRNA in human urine reveals splice variant biomarkers of muscular dystrophiesDystrophin Cardiomyopathies: Clinical Management, Molecular Pathogenesis and Evolution towards Precision MedicineCorrection of a Splicing Mutation Affecting an Unverricht-Lundborg Disease Patient by Antisense Therapy
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FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga.
description
2016 nî lūn-bûn
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2016年の論文
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2016年論文
@yue
2016年論文
@zh-hant
2016年論文
@zh-hk
2016年論文
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2016年論文
@zh-tw
2016年论文
@wuu
2016年论文
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2016年论文
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name
FDA Approves Eteplirsen for Du ...... hapter in the Eteplirsen Saga.
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FDA Approves Eteplirsen for Du ...... hapter in the Eteplirsen Saga.
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type
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FDA Approves Eteplirsen for Du ...... hapter in the Eteplirsen Saga.
@en
FDA Approves Eteplirsen for Du ...... hapter in the Eteplirsen Saga.
@nl
prefLabel
FDA Approves Eteplirsen for Du ...... hapter in the Eteplirsen Saga.
@en
FDA Approves Eteplirsen for Du ...... hapter in the Eteplirsen Saga.
@nl
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P921
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FDA Approves Eteplirsen for Du ...... hapter in the Eteplirsen Saga.
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Arthur M Krieg
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P356
10.1089/NAT.2016.0657
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2016-12-08T00:00:00Z