β-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice (Q41544464)

3 October 2017

title

β-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice (English)

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3 October 2017

E R Pozsgai

1

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3 October 2017

D A Griffin

2

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3 October 2017

K N Heller

3

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3 October 2017

J R Mendell

4

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3 October 2017

L R Rodino-Klapac

5

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3 October 2017

publication date

27 July 2015

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3 October 2017

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3 October 2017

volume

23

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3 October 2017

issue

1

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3 October 2017

page(s)

57-66

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Beta-sarcoglycan (A3b) mutations cause autosomal recessive muscular dystrophy with loss of the sarcoglycan complex

3 October 2017

cites work

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https://api.crossref.org/works/10.1038%2FGT.2015.80

Loss of the sarcoglycan complex and sarcospan leads to muscular dystrophy in beta-sarcoglycan-deficient mice

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

Disruption of the beta-sarcoglycan gene reveals pathogenetic complexity of limb-girdle muscular dystrophy type 2E

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

Genomic screening for beta-sarcoglycan gene mutations: missense mutations may cause severe limb-girdle muscular dystrophy type 2E (LGMD 2E)

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

The clinical spectrum of sarcoglycanopathies

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https://api.crossref.org/works/10.1038%2FGT.2015.80

Sarcoglycanopathies: molecular pathogenesis and therapeutic prospects

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Evidence-based guideline summary: diagnosis and treatment of limb-girdle and distal dystrophies: report of the guideline development subcommittee of the American Academy of Neurology and the practice issues review panel of the American Association o

21 January 2018

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Two siblings with limb-girdle muscular dystrophy type 2E responsive to deflazacort

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2015.80

Disruption of heart sarcoglycan complex and severe cardiomyopathy caused by beta sarcoglycan mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2015.80

Fibrosis and inflammation are greater in muscles of beta-sarcoglycan-null mouse than mdx mouse.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2015.80

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2015.80

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogates.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

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

21 January 2018

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

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

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

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

Visualization of dystrophic muscle fibers in mdx mouse by vital staining with Evans blue: evidence of apoptosis in dystrophin-deficient muscle

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

Animal models for muscular dystrophy show different patterns of sarcolemmal disruption.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.

21 January 2018

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Delivery of alpha- and beta-sarcoglycan by recombinant adeno-associated virus: efficient rescue of muscle, but differential toxicity

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D.

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

E-box sites and a proximal regulatory region of the muscle creatine kinase gene differentially regulate expression in diverse skeletal muscles and cardiac muscle of transgenic mice

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2015.80

Production and characterization of adeno-associated viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2015.80

Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2015.80

Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2015.80

The passive mechanical properties of the extensor digitorum longus muscle are compromised in 2- to 20-mo-old mdx mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2015.80

Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2015.80

Limb-girdle muscular dystrophy in the United States

21 January 2018

1 reference

12 December 2020

Heart involvement in muscular dystrophies due to sarcoglycan gene mutations

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12 December 2020

Cardiac involvement in patients with limb-girdle muscular dystrophy type 2 and Becker muscular dystrophy

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12 December 2020

LGMD2E patients risk developing dilated cardiomyopathy

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12 December 2020

Identifiers

DOI

10.1038/GT.2015.80

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Dimensions Publication ID

3 October 2017

1028733066

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