(Q37767924)

22 August 2017

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title

Gene therapy for muscle disease (English)

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22 August 2017

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Yuko Miyagoe-Suzuki

1

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22 August 2017

Shin'ichi Takeda

series ordinal

2

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22 August 2017

language of work or name

English

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publication date

24 May 2010

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Experimental Cell Research

22 August 2017

published in

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22 August 2017

volume

316

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22 August 2017

issue

18

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22 August 2017

page(s)

3087-3092

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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

22 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Three muscular dystrophies: loss of cytoskeleton-extracellular matrix linkage.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

New recombinant serotypes of AAV vectors.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Gene therapy for muscular dystrophy: current progress and future prospects.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Therapy for neuromuscular disorders

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Gene therapy progress and prospects: Duchenne muscular dystrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Recombinant adeno-associated virus type 8-mediated extensive therapeutic gene delivery into skeletal muscle of alpha-sarcoglycan-deficient mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Systemic administration of micro-dystrophin restores cardiac geometry and prevents dobutamine-induced cardiac pump failure

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

A hybrid vector system expands adeno-associated viral vector packaging capacity in a transgene-independent manner

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Construction and analysis of compact muscle-specific promoters for AAV vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Codon and mRNA Sequence Optimization of Microdystrophin Transgenes Improves Expression and Physiological Outcome in Dystrophic mdx Mice Following AAV2/8 Gene Transfer

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Stable transduction of myogenic cells with lentiviral vectors expressing a minidystrophin

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Autologous transplantation of SM/C-2.6(+) satellite cells transduced with micro-dystrophin CS1 cDNA by lentiviral vector into mdx mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Autologous transplantation of muscle precursor cells modified with a lentivirus for muscular dystrophy: human cells and primate models

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to beta-dystroglycan

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Local dystrophin restoration with antisense oligonucleotide PRO051

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Vivo-Morpholinos: a non-peptide transporter delivers Morpholinos into a wide array of mouse tissues

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

Targeting RNA: an emerging hope for treating muscular dystrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2010.05.022

10.1016/J.YEXCR.2010.05.022

7 January 2021

Identifiers

DOI

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release_zvl7efyqs5bhjhx43utmyajchu

22 August 2017

Fatcat ID

1 reference

Fatcat

https://api.fatcat.wiki/v0/release/zvl7efyqs5bhjhx43utmyajchu

mapped directly with Wikidata item

24 November 2022

based on heuristic

1 reference