Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy (Q47607001)

4 February 2018

title

Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy (English)

1 reference

4 February 2018

Toshifumi Yokota

5

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ORCID Public Data File 2021

author name string

Shouta Miyatake

1

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4 February 2018

Yoshitaka Mizobe

2

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4 February 2018

Hotake Takizawa

3

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4 February 2018

Yuko Hara

4

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4 February 2018

Shin'ichi Takeda

6

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4 February 2018

Yoshitsugu Aoki

7

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4 February 2018

language of work or name

English

0 references

publication date

1 January 2018

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4 February 2018

Methods in Molecular Biology

number of pages

19

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based on heuristic

inferred from page(s)

published in

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4 February 2018

volume

1687

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4 February 2018

page(s)

123-141

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4 February 2018

In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse

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Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

21 January 2018

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RNA therapeutics: beyond RNA interference and antisense oligonucleotides

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Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics.

21 January 2018

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Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles

21 January 2018

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Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

21 January 2018

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Antisense oligonucleotide drugs for Duchenne muscular dystrophy: how far have we come and what does the future hold?

21 January 2018

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New developments in exon skipping and splice modulation therapies for neuromuscular diseases

21 January 2018

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Eteplirsen therapy for Duchenne muscular dystrophy: skipping to the front of the line.

21 January 2018

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Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule

21 January 2018

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Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

21 January 2018

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Dermcidin: a skeletal muscle myokine modulating cardiomyocyte survival and infarct size after coronary artery ligation.

21 January 2018

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Targeted Disruption of Exon 52 in the Mouse Dystrophin Gene Induced Muscle Degeneration Similar to That Observed in Duchenne Muscular Dystrophy

21 January 2018

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Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice.

21 January 2018

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Dp260 disrupted mice revealed prolonged implicit time of the b-wave in ERG and loss of accumulation of beta-dystroglycan in the outer plexiform layer of the retina

21 January 2018

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Absent pituitary gland in two brothers with an oral-facial-digital syndrome resembling OFDS II and VI: a new type of OFDS?

21 January 2018

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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

21 January 2018

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Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of exon skipping therapy.

21 January 2018

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Muscle plasma membrane changes in dystrophin gene exon 52 knockout mouse

21 January 2018

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Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells.

21 January 2018

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TRPC1 binds to caveolin-3 and is regulated by Src kinase - role in Duchenne muscular dystrophy

21 January 2018

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Mutation types and aging differently affect revertant fiber expansion in dystrophic mdx and mdx52 mice

21 January 2018

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A new model mouse for Duchenne muscular dystrophy produced by 2.4 Mb deletion of dystrophin gene using Cre-loxP recombination system

21 January 2018

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FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

21 January 2018

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Generation and Characterization of Transgenic Mice with the Full-length HumanDMDGene

21 January 2018

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10.1007/978-1-4939-7374-3_9

21 January 2018

Identifiers

DOI

1 reference

4 February 2018

1 reference

4 February 2018