Dystrophin quantification: Biological and translational research implications. - Wikidata - awgldk - TriplyDB

Dystrophin quantification: Biological and translational research implications.

Dystrophin quantification: Biological and translational research implications.

http://www.wikidata.org/entity/Q34591203

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The importance of genetic diagnosis for Duchenne muscular dystrophy Genetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy.Digital Droplet PCR for the Absolute Quantification of Exon Skipping Induced by Antisense Oligonucleotides in (Pre-)Clinical Development for Duchenne Muscular Dystrophy.Skeletal muscle water T2 as a biomarker of disease status and exercise effects in patients with Duchenne muscular dystrophy.Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.Dystrophin Distribution and Expression in Human and Experimental Temporal Lobe Epilepsy.Exon skipping: a first in class strategy for Duchenne muscular dystrophy.Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies Proceedings of the 2017 National Toxicology Program Satellite Symposium.Myoblots: dystrophin quantification by in-cell western assay for a streamlined development of Duchenne muscular dystrophy (DMD) treatments.Duchenne and Becker Muscular Dystrophies: A Review of Animal Models, Clinical End Points, and Biomarker Quantification.A novel high-throughput immunofluorescence analysis method for quantifying dystrophin intensity in entire transverse sections of Duchenne muscular dystrophy muscle biopsy samples.Use of capillary Western immunoassay (Wes) for quantification of dystrophin levels in skeletal muscle of healthy controls and individuals with Becker and Duchenne muscular dystrophy.Assisted delivery of antisense therapeutics in animal models of heritable neurodegenerative and neuromuscular disorders: a systematic review and meta-analysis.

P2860

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P2860

Dystrophin quantification: Biological and translational research implications.

http://www.wikidata.org/entity/Q34591203

description

2014 nî lūn-bûn

@nan

2014 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած

@hyw

2014 թվականի հոտեմբերին հրատարակված գիտական հոդված

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2014年の論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年论文

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name

Dystrophin quantification: Biological and translational research implications.

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Dystrophin quantification: Biological and translational research implications.

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type

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Dystrophin quantification: Biological and translational research implications.

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Dystrophin quantification: Biological and translational research implications.

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Dystrophin quantification: Biological and translational research implications.

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Dystrophin quantification: Biological and translational research implications.

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Dystrophin quantification: Biological and translational research implications.

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Adeline Vulin

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Afrodite Lourbakos

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Caroline A Sewry

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Francesco Muntoni

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Giles Campion

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Jennifer E Morgan

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Laura E Taylor

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Lucy Feng

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Matt Henderson

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Maud Beuvin

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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

Statistical methods for assessing agreement between two methods of clinical measurement

A sensitive, reproducible and objective immunofluorescence analysis method of dystrophin in individual fibers in samples from patients with duchenne muscular dystrophy

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.

The ZZ domain of dystrophin in DMD: making sense of missense mutations.

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

Dystrophin and mutations: one gene, several proteins, multiple phenotypes.

Internal deletion compromises the stability of dystrophin

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83

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Virginia Arechavala-Gomeza

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