Safety and in vivo expression of a GNE-transgene: a novel treatment approach for hereditary inclusion body myopathy-2. (Q37378486)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19838336 AND SRC:MED&resulttype=core&format=json

10 February 2020

title

Safety and in vivo expression of a GNE-transgene: a novel treatment approach for hereditary inclusion body myopathy-2 (English)

1 reference

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10 February 2020

13

John Nemunaitis

1 reference

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10 February 2020

author name string

Anagha P Phadke

1

1 reference

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10 February 2020

Chris Jay

2

1 reference

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10 February 2020

Salina J Chen

3

1 reference

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10 February 2020

Courtney Haddock

4

1 reference

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10 February 2020

Zhaohui Wang

5

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10 February 2020

Yang Yu

6

1 reference

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10 February 2020

Derek Nemunaitis

7

1 reference

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10 February 2020

Gregory Nemunaitis

8

1 reference

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10 February 2020

Nancy S Templeton

9

1 reference

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10 February 2020

Neil Senzer

10

1 reference

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10 February 2020

Phillip B Maples

11

1 reference

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10 February 2020

Alex W Tong

12

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10 February 2020

publication date

8 May 2009

1 reference

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Gene Regulation and Systems Biology

10 February 2020

published in

1 reference

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10 February 2020

volume

3

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10 February 2020

page(s)

89-101

1 reference

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Preclinical assessment of wt GNE gene plasmid for management of hereditary inclusion body myopathy 2 (HIBM2)

10 February 2020

cites work

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6 September 2017

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A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs.

6 September 2017

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Mutation in the key enzyme of sialic acid biosynthesis causes severe glomerular proteinuria and is rescued by N-acetylmannosamine

6 September 2017

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Intravenous immune globulin in hereditary inclusion body myopathy: a pilot study

6 September 2017

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Reduced sialylation status in UDP-N-acetylglucosamine-2-epimerase/N-acetylmannosamine kinase (GNE)-deficient mice

6 September 2017

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Distal myopathy with rimmed vacuoles is allelic to hereditary inclusion body myopathy

6 September 2017

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Four novel mutations associated with autosomal recessive inclusion body myopathy (MIM: 600737)

6 September 2017

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Sialylation is essential for early development in mice

6 September 2017

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The UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase gene is mutated in recessive hereditary inclusion body myopathy

6 September 2017

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Nonviral vectors in the new millennium: delivery barriers in gene transfer

6 September 2017

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Characterizing glycosylation pathways

6 September 2017

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Comprehensive analysis of the acute toxicities induced by systemic administration of cationic lipid:plasmid DNA complexes in mice

6 September 2017

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

6 September 2017

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New directions in liposome gene delivery

6 September 2017

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Primary structure and expression analysis of human UDP-N-acetyl-glucosamine-2-epimerase/N-acetylmannosamine kinase, the bifunctional enzyme in neuraminic acid biosynthesis

6 September 2017

1 reference

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UDP-GlcNAc 2-epimerase: a regulator of cell surface sialylation

6 September 2017

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Tissue expression and amino acid sequence of murine UDP-N-acetylglucosamine-2-epimerase/N-acetylmannosamine kinase

6 September 2017

1 reference

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A bifunctional enzyme catalyzes the first two steps in N-acetylneuraminic acid biosynthesis of rat liver. Purification and characterization of UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase

6 September 2017

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In vivo comparative study of lipid/DNA complexes with different in vitro serum stability: effects on biodistribution and tumor accumulation

6 September 2017

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GNE protein expression and subcellular distribution are unaltered in HIBM.

14 September 2017

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Complex formation with plasmid DNA increases the cytotoxicity of cationic liposomes

14 September 2017

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Local and systemic inhibition of lung tumor growth after nanoparticle-mediated mda-7/IL-24 gene delivery.

14 September 2017

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The homozygous M712T mutation of UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase results in reduced enzyme activities but not in altered overall cellular sialylation in hereditary inclusion body myopathy

14 September 2017

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14 September 2017

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Characterization of hereditary inclusion body myopathy myoblasts: possible primary impairment of apoptotic events

26 June 2018

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Influence of UDP-GlcNAc 2-epimerase/ManNAc kinase mutant proteins on hereditary inclusion body myopathy.

2 September 2018

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Mutation analysis of the GNE gene in Korean patients with distal myopathy with rimmed vacuoles

2 September 2018

1 reference

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Intravenous delivery of liposome-mediated nonviral DNA is less toxic than intraperitoneal delivery in mice

2 September 2018

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No overall hyposialylation in hereditary inclusion body myopathy myoblasts carrying the homozygous M712T GNE mutation

2 September 2018

1 reference

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Hypoglycosylation of alpha-dystroglycan in patients with hereditary IBM due to GNE mutations.

2 September 2018

1 reference

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Reduction of UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase activity and sialylation in distal myopathy with rimmed vacuoles

2 September 2018

1 reference

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A novel homozygous missense mutation in the GNE gene of a patient with quadriceps-sparing hereditary inclusion body myopathy associated with muscle inflammation

2 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2758285

Optimization of gene expression in nonactivated circulating lymphocytes

2 September 2018

1 reference

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Mutations spectrum of GNE in hereditary inclusion body myopathy sparing the quadriceps.

2 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2758285

Contribution of plasmid DNA to hepatotoxicity after systemic administration of lipoplexes

2 September 2018

1 reference

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Successful treatment of primary and disseminated human lung cancers by systemic delivery of tumor suppressor genes using an improved liposome vector

2 September 2018

1 reference

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Liposome-mediated, nonviral gene transfer induces a systemic inflammatory response which can exacerbate pre-existing inflammation

2 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2758285

A Gne knockout mouse expressing human GNE D176V mutation develops features similar to distal myopathy with rimmed vacuoles or hereditary inclusion body myopathy

2 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2758285

Mutation analysis of theGNE gene in distal myopathy with rimmed vacuoles (DMRV) patients in Thailand

3 December 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2758285

Back to basics. Administering i.m. injections the right way

3 December 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/19838336

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

NCAM is hyposialylated in hereditary inclusion body myopathy due to GNE mutations

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/19838336

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.4137/GRSB.S2210

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19838336 AND SRC:MED&resulttype=core&format=json

10 February 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19838336 AND SRC:MED&resulttype=core&format=json

10 February 2020

PubMed publication ID

19838336

1 reference