NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy

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NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy

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wetenschappelijk artikel

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наукова стаття, опублікована у вересні 2018

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NS-065/NCNP-01: An Antisense O ...... in Duchenne Muscular Dystrophy

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NS-065/NCNP-01: An Antisense O ...... in Duchenne Muscular Dystrophy

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NS-065/NCNP-01: An Antisense O ...... in Duchenne Muscular Dystrophy

@en

NS-065/NCNP-01: An Antisense O ...... in Duchenne Muscular Dystrophy

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NS-065/NCNP-01: An Antisense O ...... in Duchenne Muscular Dystrophy

@en

NS-065/NCNP-01: An Antisense O ...... in Duchenne Muscular Dystrophy

@nl

P1476

NS-065/NCNP-01: An Antisense O ...... in Duchenne Muscular Dystrophy

@en

P2093

Hidetoshi Kitagawa

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Hirofumi Komaki

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Kazuchika Takagaki

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Naoki Watanabe

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Satoru Masuda

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Shin'ichi Takeda

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Takashi Saito

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Tetsuya Nagata

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Youhei Satou

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P2860

Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient

Dynamics of co-transcriptional pre-mRNA folding influences the induction of dystrophin exon skipping by antisense oligonucleotides.

In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.

A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy.

The influence of antisense oligonucleotide length on dystrophin exon skipping.

The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations.

Pharmacokinetics and biodistribution of phosphorodiamidate morpholino antisense oligomers.

Therapeutic advances in muscular dystrophy

Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites.

Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene.

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442-449

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P31

scholarly article

P356

10.1016/J.OMTN.2018.09.017

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P407

English

P478

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P577

2018-09-27T00:00:00Z

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P698

30388618

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P921

drug discovery

molecular medicine

P932

6202794

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