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Splice-switching antisense oligonucleotides as therapeutic drugs

Splice-switching antisense oligonucleotides as therapeutic drugs

http://www.wikidata.org/entity/Q37211004

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Antisense oligonucleotides delivered to the amniotic cavity in utero modulate gene expression in the postnatal mouse.GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells.Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease Development of an orally available inhibitor of CLK1 for skipping a mutated dystrophin exon in Duchenne muscular dystrophy The U2AF1S34F mutation induces lineage-specific splicing alterations in myelodysplastic syndromes.LOX-1 and Its Splice Variants: A New Challenge for Atherosclerosis and Cancer-Targeted Therapies.Synthesis and cellular activity of stereochemically-pure 2'-O-(2-methoxyethyl)-phosphorothioate oligonucleotides.Mutations of Pre-mRNA Splicing Regulatory Elements: Are Predictions Moving Forward to Clinical Diagnostics?Alternative splicing: the pledge, the turn, and the prestige : The key role of alternative splicing in human biological systems.RNA splicing during terminal erythropoiesis.Nucleic Acid Templated Reactions for Chemical Biology.Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases.The emerging role of alternative splicing in senescence and aging.Synthesis of oligonucleotides on a soluble support.The role of alternative splicing in cancer.RNA splicing process analysis for identifying antisense oligonucleotide inhibitors with padlock probe-based isothermal amplification Emerging roles of RNA-binding proteins in diabetes and their therapeutic potential in diabetic complications.Antisense oligonucleotides: the next frontier for treatment of neurological disorders.Glycogen Reduction in Myotubes of Late-Onset Pompe Disease Patients Using Antisense Technology.Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs.Rescue of Outer Hair Cells with Antisense Oligonucleotides in Usher Mice Is Dependent on Age of Treatment.Therapeutic Applications of Targeted Alternative Splicing to Cancer Treatment.Impact, Characterization, and Rescue of Pre-mRNA Splicing Mutations in Lysosomal Storage Disorders.The potential of antisense oligonucleotide therapies for inherited childhood lung diseases.Nusinersen for the treatment of spinal muscular atrophy.Rescue of peripheral vestibular function in Usher syndrome mice using a splice-switching antisense oligonucleotide.Alternative-splicing defects in cancer: Splicing regulators and their downstream targets, guiding the way to novel cancer therapeutics.Targeting Amyloid-β Precursor Protein, APP, Splicing with Antisense Oligonucleotides Reduces Toxic Amyloid-β Production.Editorial: Nucleic Acids Research and Nucleic Acid Therapeutics.Antisense Phosphorodiamidate Morpholino Oligomers as Novel Antiviral Compounds.Alternative mRNA Splicing in the Pathogenesis of Obesity.Intronic PAH gene mutations cause a splicing defect by a novel mechanism involving U1snRNP binding downstream of the 5' splice site.Targeting Splicing in Prostate Cancer.Synthesis, Affinity for Complementary RNA and DNA, and Enzymatic Stability of Triazole-Linked Locked Nucleic Acids (t-LNAs).Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers.Morpholino Antisense Oligomers as a Potential Therapeutic Option for the Correction of Alternative Splicing in PMD, SPG2, and HEMS.RNA Therapeutics in Cardiovascular Precision Medicine Cationic Oligospermine-Oligonucleotide Conjugates Provide Carrier-free Splice Switching in Monolayer Cells and Spheroids

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P2860

Splice-switching antisense oligonucleotides as therapeutic drugs

http://www.wikidata.org/entity/Q37211004

description

article científic

@ca

article scientifique

@fr

articolo scientifico

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artigo científico

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bilimsel makale

@tr

scientific article published on 10 June 2016

@en

vedecký článok

@sk

vetenskaplig artikel

@sv

videnskabelig artikel

@da

vědecký článek

@cs

name

Splice-switching antisense oligonucleotides as therapeutic drugs

@en

Splice-switching antisense oligonucleotides as therapeutic drugs.

@nl

type

label

Splice-switching antisense oligonucleotides as therapeutic drugs

@en

Splice-switching antisense oligonucleotides as therapeutic drugs.

@nl

prefLabel

Splice-switching antisense oligonucleotides as therapeutic drugs

@en

Splice-switching antisense oligonucleotides as therapeutic drugs.

@nl

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Splice-switching antisense oligonucleotides as therapeutic drugs

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Mallory A Havens

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Michelle L Hastings

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P2860

Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Splicing regulation: from a parts list of regulatory elements to an integrated splicing code

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy

The Clothes Make the mRNA: Past and Present Trends in mRNP Fashion

The 3.8 Å structure of the U4/U6.U5 tri-snRNP: Insights into spliceosome assembly and catalysis

Defective control of pre-messenger RNA splicing in human disease

Spinal Muscular Atrophy

A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy

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6549-6563

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scholarly article

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10.1093/NAR/GKW533

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14

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Michelle L Hastings

Mallory A Havens

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2016-08-19T00:00:00Z

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regulation of gene expression

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5001604

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