Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy - Wikidata - awgldk - TriplyDB

Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy

Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy

http://www.wikidata.org/entity/Q36714202

about

Molecular and cell-based therapies for muscle degenerations: a road under construction Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials The novel Cln1(R151X) mouse model of infantile neuronal ceroid lipofuscinosis (INCL) for testing nonsense suppression therapy Current understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophy Evaluation of Aminoglycoside and Non-Aminoglycoside Compounds for Stop-Codon Readthrough Therapy in Four Lysosomal Storage Diseases Postnatal manipulation of Pax6 dosage reverses congenital tissue malformation defects.Translational read-through as an alternative approach for ocular gene therapy of retinal dystrophies caused by in-frame nonsense mutations.Chronic inflammation and cancer: potential chemoprevention through nuclear factor kappa B and p53 mutual antagonism Toward a rationale for the PTC124 (Ataluren) promoted readthrough of premature stop codons: a computational approach and GFP-reporter cell-based assay Ataluren treatment of patients with nonsense mutation dystrophinopathy.Nonsense-mediated decay in genetic disease: friend or foe?Feasibility of resistance training in adult McArdle patients: clinical outcomes and muscle strength and mass benefits.A new series of small molecular weight compounds induce read through of all three types of nonsense mutations in the ATM gene.High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.Aquapuncture Using Stem Cell Therapy to Treat Mdx Mice.Jagged 1 Rescues the Duchenne Muscular Dystrophy Phenotype.Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice.Premature termination codon read-through in the ABCC6 gene: potential treatment for pseudoxanthoma elasticum.Circulating Biomarkers for Duchenne Muscular Dystrophy.Ataluren as an agent for therapeutic nonsense suppression.Nonsense Suppression as an Approach to Treat Lysosomal Storage Diseases.Update on the treatment of Duchenne muscular dystrophy.New developments in the use of gene therapy to treat Duchenne muscular dystrophy.Treatment of dystrophin cardiomyopathies.McArdle disease: a unique study model in sports medicine.Drug Discovery of Therapies for Duchenne Muscular Dystrophy.Targeting Nonsense Mutations in Diseases with Translational Read-Through-Inducing Drugs (TRIDs).The role of nonsense-mediated decay in neuronal ceroid lipofuscinosis.Efficacy of Postnatal In Vivo Nonsense Suppression Therapy in a Pax6 Mouse Model of Aniridia.Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches.Optimized approach for the identification of highly efficient correctors of nonsense mutations in human diseases.ECM-Related Myopathies and Muscular Dystrophies: Pros and Cons of Protein Therapies.Therapeutic effect of prenatal alkalization and PTC124 in Na(+)/HCO3(-) cotransporter 1 p.W516* knock-in mice.Ex vivo gene editing of the dystrophin gene in muscle stem cells mediated by peptide nucleic acid single stranded oligodeoxynucleotides induces stable expression of dystrophin in a mouse model for Duchenne muscular dystrophy.Aminoglycoside-mediated promotion of translation readthrough occurs through a non-stochastic mechanism that competes with translation termination.The effect of PTC124 on choroideremia fibroblasts and iPSC-derived RPE raises considerations for therapy.Advances in therapeutic use of a drug-stimulated translational readthrough of premature termination codons.

P2860

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P2860

Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy

http://www.wikidata.org/entity/Q36714202

description

2012 nî lūn-bûn

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2012年の論文

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2012年論文

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2012年論文

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2012年論文

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2012年論文

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2012年論文

@zh-tw

2012年论文

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2012年论文

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2012年论文

@zh-cn

name

Read-through compound 13 resto ...... or Duchenne muscular dystrophy

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Read-through compound 13 resto ...... or Duchenne muscular dystrophy

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type

label

Read-through compound 13 resto ...... or Duchenne muscular dystrophy

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Read-through compound 13 resto ...... or Duchenne muscular dystrophy

@en

prefLabel

Read-through compound 13 resto ...... or Duchenne muscular dystrophy

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Read-through compound 13 resto ...... or Duchenne muscular dystrophy

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Q36714202-161F710A-330F-4EBB-9D77-1A0C063E3700

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P2860

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Human Molecular Genetics

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Read-through compound 13 resto ...... or Duchenne muscular dystrophy

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P2093

Carmen Bertoni

http://www.w3.org/2001/XMLSchema#string

Gregory Khitrov

http://www.w3.org/2001/XMLSchema#string

Jin-Mo Ku

http://www.w3.org/2001/XMLSchema#string

Michael E Jung

http://www.w3.org/2001/XMLSchema#string

Olga Prikhodko

http://www.w3.org/2001/XMLSchema#string

Refik Kayali

http://www.w3.org/2001/XMLSchema#string

P2860

Osteopontin promotes fibrosis in dystrophic mouse muscle by modulating immune cell subsets and intramuscular TGF-beta

Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124

The nonsense-mediated decay RNA surveillance pathway

Gentamicin-mediated suppression of Hurler syndrome stop mutations restores a low level of alpha-L-iduronidase activity and reduces lysosomal glycosaminoglycan accumulation.

Development and validation of an HPLC method for quantitation of BA-TPQ, a novel iminoquinone anticancer agent, and an initial pharmacokinetic study in mice

Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice

Testing of SHIRPA, a mouse phenotypic assessment protocol, on Dmd(mdx) and Dmd(mdx3cv) dystrophin-deficient mice.

Preclinical drug trials in the mdx mouse: assessment of reliable and sensitive outcome measures.

Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.

PTC124 targets genetic disorders caused by nonsense mutations.

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4007-4020

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scholarly article

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10.1093/HMG/DDS223

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18

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21

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2012-06-12T00:00:00Z

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22692682

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3607466

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