Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy.

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Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy.

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2018 nî lūn-bûn

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2018年の論文

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2018年学术文章

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2018年学术文章

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2018年学术文章

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2018年学术文章

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2018年學術文章

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name

Peptide-conjugated phosphodiam ...... f Duchenne muscular dystrophy.

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Peptide-conjugated phosphodiam ...... f Duchenne muscular dystrophy.

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label

Peptide-conjugated phosphodiam ...... f Duchenne muscular dystrophy.

@en

Peptide-conjugated phosphodiam ...... f Duchenne muscular dystrophy.

@nl

prefLabel

Peptide-conjugated phosphodiam ...... f Duchenne muscular dystrophy.

@en

Peptide-conjugated phosphodiam ...... f Duchenne muscular dystrophy.

@nl

P1476

Peptide-conjugated phosphodiam ...... of Duchenne muscular dystrophy

@en

P2093

Caroline Godfrey

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Elizabeth Greally

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Graham McClorey

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Guy A MacGowan

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Liz O'Donovan

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Mike J Gait

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Raquel Manzano

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Thibault Coursindel

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Volker W Straub

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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

Population frequencies of inherited neuromuscular diseases--a world survey

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

Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.

A human-specific deletion in mouse Cmah increases disease severity in the mdx model of Duchenne muscular dystrophy

Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene.

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

Physiological characterization of muscle strength with variable levels of dystrophin restoration in mdx mice following local antisense therapy.

One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice.

The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion

P304

e0198897

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scholarly article

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10.1371/JOURNAL.PONE.0198897

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P407

English

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P478

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P50

Corinne A Betts

Matthew J. Wood

Alison M Blain

P577

2018-06-18T00:00:00Z

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P698

29912990

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P932

6005479

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