Amelioration of laminin-alpha2-deficient congenital muscular dystrophy by somatic gene transfer of miniagrin (Q33922811)

30 July 2017

Amelioration of laminin-alpha2-deficient congenital muscular dystrophy by somatic gene transfer of miniagrin (English)

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congenital muscular dystrophy

30 July 2017

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1 reference

Chunping Qiao

1

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30 July 2017

Jianbin Li

2

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30 July 2017

Tong Zhu

3

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30 July 2017

Romesh Draviam

4

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30 July 2017

Simon Watkins

5

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30 July 2017

Xiaojing Ye

6

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30 July 2017

Chunlian Chen

7

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30 July 2017

Juan Li

8

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30 July 2017

Xiao Xiao

9

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30 July 2017

language of work or name

English

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publication date

15 August 2005

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30 July 2017

6

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Proceedings of the National Academy of Sciences of the United States of America

inferred from page(s)

published in

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30 July 2017

102

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30 July 2017

34

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30 July 2017

11999-12004

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Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice

30 July 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

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

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

The basement membrane/basal lamina of skeletal muscle

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Muscle-specific expression of insulin-like growth factor I counters muscle decline in mdx mice

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

An agrin minigene rescues dystrophic symptoms in a mouse model for congenital muscular dystrophy.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

The dystroglycan complex is necessary for stabilization of acetylcholine receptor clusters at neuromuscular junctions and formation of the synaptic basement membrane

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Identification and characterization of 5' extension of mammalian agrin cDNA, the exons and the promoter sequences

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

A novel laminin alpha2 isoform in severe laminin alpha2 deficient congenital muscular dystrophy

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Rescue of skeletal muscles of gamma-sarcoglycan-deficient mice with adeno-associated virus-mediated gene transfer.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Form and function: the laminin family of heterotrimers

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

AAV vectors: is clinical success on the horizon?

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Full functional rescue of a complete muscle (TA) in dystrophic hamsters by adeno-associated virus vector-directed gene therapy

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Gene therapy vectors based on adeno-associated virus type 1.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Assignment of the muscle-eye-brain disease gene to 1p32-p34 by linkage analysis and homozygosity mapping

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Merosin-deficient congenital muscular dystrophy. Partial genetic correction in two mouse models

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

An ancient retrotransposal insertion causes Fukuyama-type congenital muscular dystrophy

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Agrin is a high-affinity binding protein of dystroglycan in non-muscle tissue

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Distribution and function of laminins in the neuromuscular system of developing, adult, and mutant mice

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Merosin/laminin-2 and muscular dystrophy

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Merosin and laminin in myogenesis; specific requirement for merosin in myotube stability and survival

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Differential heparin inhibition of skeletal muscle alpha-dystroglycan binding to laminins

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Dystroglycan-alpha, a dystrophin-associated glycoprotein, is a functional agrin receptor

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Defective muscle basement membrane and lack of M-laminin in the dystrophic dy/dy mouse

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Deficiency of merosin in dystrophic dy mice and genetic linkage of laminin M chain gene to dy locus

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Merosin, a protein specific for basement membranes of Schwann cells, striated muscle, and trophoblast, is expressed late in nerve and muscle development

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Primary structure of dystrophin-associated glycoproteins linking dystrophin to the extracellular matrix

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

A novel laminin 2 isoform in severe laminin 2 deficient congenital muscular dystrophy

5 July 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

The role of laminins in basement membrane function

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

Expression of laminin alpha1, alpha2, alpha4, and alpha5 chains, fibronectin, and tenascin-C in skeletal muscle of dystrophic 129ReJ dy/dy mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

Combined maternal and congenital myotonic dystrophy managed by a multidisciplinary team

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

Clinical gene transfer studies for hemophilia B.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

Adeno-associated virus vector-mediated minidystrophin gene therapy improves dystrophic muscle contractile function in mdx mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

Laminin alpha1 chain reduces muscular dystrophy in laminin alpha2 chain deficient mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

Regulation of integrin growth factor interactions in oligodendrocytes by lipid raft microdomains

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1189311

Laminin α2 deficiency and muscular dystrophy; genotype-phenotype correlation in mutant mice

29 October 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0502137102

Muscular dystrophy with laminin deficiency decreases the content of butyrylcholinesterase tetramers in sciatic nerves of Lama2dy mice

21 January 2018

1 reference

12 December 2020

Collagen types in neuromuscular diseases

1 reference

12 December 2020

Localization of merosin in the normal human brain: implications for congenital muscular dystrophy with merosin deficiency

1 reference

12 December 2020

Is there selection in favour of heterozygotes in families with merosin-deficient congenital muscular dystrophy?

1 reference

12 December 2020

T-cell-dependent fibrosis in the mdx dystrophic mouse

1 reference

12 December 2020

Laminin alpha 2 (merosin)-deficient muscular dystrophy and demyelinating neuropathy in two cats

1 reference

12 December 2020

The muscular dystrophies

1 reference

12 December 2020

Merosin-deficient congenital muscular dystrophy, autosomal recessive (MDC1A, MIM#156225, LAMA2 gene coding for alpha2 chain of laminin)

1 reference

12 December 2020

Identifiers

DOI

10.1073/PNAS.0502137102

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30 July 2017

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30 July 2017

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