The effects of low levels of dystrophin on mouse muscle function and pathology - sprookjes - yvandenbroek - TriplyDB

The effects of low levels of dystrophin on mouse muscle function and pathology

The effects of low levels of dystrophin on mouse muscle function and pathology

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Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy P2RX7 purinoceptor: a therapeutic target for ameliorating the symptoms of duchenne muscular dystrophy Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice Effective dystrophin restoration by a novel muscle-homing peptide-morpholino conjugate in dystrophin-deficient mdx mice.Conserved regions of the DMD 3' UTR regulate translation and mRNA abundance in cultured myotubes Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders.Assessing functional performance in the mdx mouse model Aquapuncture Using Stem Cell Therapy to Treat Mdx Mice.How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse Disease-proportional proteasomal degradation of missense dystrophins Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy.The lack of the Celf2a splicing factor converts a Duchenne genotype into a Becker phenotype.TNF-α-Induced microRNAs Control Dystrophin Expression in Becker Muscular Dystrophy In vivo gene editing in dystrophic mouse muscle and muscle stem cells.Genomic removal of a therapeutic mini-dystrophin gene from adult mice elicits a Duchenne muscular dystrophy-like phenotype Comparison of Experimental Protocols of Physical Exercise for mdx Mice and Duchenne Muscular Dystrophy Patients.Mild Aerobic Exercise Training Hardly Affects the Diaphragm of mdx Mice.Dystrophin: The dead calm of a dogma.Uniform low-level dystrophin expression in the heart partially preserved cardiac function in an aged mouse model of Duchenne cardiomyopathy.Functional correction of dystrophin actin binding domain mutations by genome editing.Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models.Adaptive Immune Response Impairs the Efficacy of Autologous Transplantation of Engineered Stem Cells in Dystrophic Dogs.Mouse models of two missense mutations in actin binding domain 1 of dystrophin associated with Duchenne or Becker muscular dystrophy.Cognitive flexibility deficits in a mouse model for the absence of full-length dystrophin.What can we learn from assisted bicycle training in a girl with dystrophinopathy? A case study.Accurate Dystrophin Quantification in Mouse Tissue; Identification of New and Evaluation of Existing Methods.A dystrophic Duchenne mouse model for testing human antisense oligonucleotides.Therapeutic effects of exon skipping and losartan on skeletal muscle of mdx mice.Dystrophin levels and clinical severity in Becker muscular dystrophy patients.Influence of full-length dystrophin on brain volumes in mouse models of Duchenne muscular dystrophy.Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy Alterations in the muscle force transfer apparatus in aged rats during unloading and reloading: impact of microRNA-31

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The effects of low levels of dystrophin on mouse muscle function and pathology

http://www.wikidata.org/entity/Q34168463

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2012 nî lūn-bûn

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2012 թուականի Փետրուարին հրատարակուած գիտական յօդուած

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2012 թվականի փետրվարին հրատարակված գիտական հոդված

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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

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2012年论文

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The effects of low levels of dystrophin on mouse muscle function and pathology

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The effects of low levels of dystrophin on mouse muscle function and pathology

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The effects of low levels of dystrophin on mouse muscle function and pathology

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The effects of low levels of dystrophin on mouse muscle function and pathology

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Ella van Huizen

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Johan T den Dunnen

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Maaike van Putten

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Maarten van Iterson

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Margriet Hulsker

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Peter Admiraal

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Sandra H van Heiningen

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Vishna Devi Nadarajah

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P2860

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

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

Muscle regeneration in dystrophin-deficient mdx mice studied by gene expression profiling

Assumption-free analysis of quantitative real-time polymerase chain reaction (PCR) data

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

Blastocyst injection of wild type embryonic stem cells induces global corrections in mdx mice.

Muscular dystrophy in girls with X;autosome translocations.

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.

Xist gene regulation at the onset of X inactivation.

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