Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers. (Q39044313)

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29 February 2020

title

Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers (English)

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29 February 2020

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29 February 2020

Matthew J. Wood

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29 February 2020

Cyrille Vaillend

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29 February 2020

Christian J. Leumann

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Kay E Davies

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4

Samir El Andaloussi

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3

Arran Babbs

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29 February 2020

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author name string

Graziella Griffith

2

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29 February 2020

Kariem Ezzat

5

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29 February 2020

Aurélie Avril

6

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29 February 2020

Branislav Dugovic

7

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29 February 2020

Rémi Chaussenot

8

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29 February 2020

Arnaud Ferry

9

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Thomas Voit

10

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Helge Amthor

11

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29 February 2020

Claudia Bühr

12

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29 February 2020

Stefan Schürch

13

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29 February 2020

Luis Garcia

18

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language of work or name

English

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publication date

2 February 2015

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29 February 2020

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29 February 2020

volume

21

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issue

3

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29 February 2020

page(s)

270-275

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Systemic administration of PRO051 in Duchenne's muscular dystrophy

29 February 2020

cites work

1 reference

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

21 January 2018

1 reference

Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals

21 January 2018

1 reference

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

21 January 2018

1 reference

Cognitive impairment in Duchenne muscular dystrophy.

21 January 2018

1 reference

Eteplirsen for the treatment of Duchenne muscular dystrophy

21 January 2018

1 reference

What Can We Learn From Clinical Trials of Exon Skipping for DMD?

21 January 2018

1 reference

Emergent dilated cardiomyopathy caused by targeted repair of dystrophic skeletal muscle

21 January 2018

1 reference

Watson-Crick base-pairing properties of tricyclo-DNA.

21 January 2018

1 reference

Position-dependent effects on stability in tricyclo-DNA modified oligonucleotide duplexes

21 January 2018

1 reference

Antisense properties of tricyclo-DNA

21 January 2018

1 reference

Tricyclo-DNA containing oligonucleotides as steric block inhibitors of human immunodeficiency virus type 1 tat-dependent trans-activation and HIV-1 infectivity

21 January 2018

1 reference

TricycloDNA-modified oligo-2'-deoxyribonucleotides reduce scavenger receptor B1 mRNA in hepatic and extra-hepatic tissues--a comparative study of oligonucleotide length, design and chemistry.

21 January 2018

1 reference

Understanding biophysicochemical interactions at the nano-bio interface

21 January 2018

1 reference

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

21 January 2018

1 reference

In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

21 January 2018

1 reference

Quality of life, physical disability, and respiratory impairment in Duchenne muscular dystrophy

21 January 2018

1 reference

Dystrophin-deficient cardiomyopathy in mouse: expression of Nox4 and Lox are associated with fibrosis and altered functional parameters in the heart

21 January 2018

1 reference

A deficit of brain dystrophin impairs specific amygdala GABAergic transmission and enhances defensive behaviour in mice

21 January 2018

1 reference

Reduction of abnormal behavioral response to brief restraint by information from other mice in dystrophin-deficient mdx mice

21 January 2018

1 reference

Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy

21 January 2018

1 reference

Selection of oligonucleotide aptamers with enhanced uptake and activation of human leukemia B cells

21 January 2018

1 reference

Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice

21 January 2018

1 reference

Dose-dependent pharmacokinetic profiles of 2'-O-methyl phosphorothioate antisense oligonucleotidesin mdx mice

21 January 2018

1 reference

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping

21 January 2018

1 reference

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping

21 January 2018

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12 December 2020

The muscular dystrophies

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12 December 2020

Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice.

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12 December 2020

Dystrophins, utrophins, and associated scaffolding complexes: role in mammalian brain and implications for therapeutic strategies

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12 December 2020

Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy

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12 December 2020

Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: a model for Duchenne muscular dystrophy

1 reference

12 December 2020

The molecular basis of muscular dystrophy in the mdx mouse: a point mutation

1 reference

12 December 2020

Evolution of the mdx mouse cardiomyopathy: physiological and morphological findings

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12 December 2020