c9RAN translation: a potential therapeutic target for the treatment of amyotrophic lateral sclerosis and frontotemporal dementia (Q48026873)

7 February 2018

editorial

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title

c9RAN translation: a potential therapeutic target for the treatment of amyotrophic lateral sclerosis and frontotemporal dementia (English)

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7 February 2018

amyotrophic lateral sclerosis and frontotemporal dementia

main subject

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amyotrophic lateral sclerosis

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frontotemporal dementia

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3

Leonard Petrucelli

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7 February 2018

Tania F Gendron

1

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7 February 2018

Danielle M Cosio

series ordinal

2

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7 February 2018

language of work or name

English

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publication date

12 July 2013

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7 February 2018

Expert Opinion on Therapeutic Targets

published in

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7 February 2018

volume

17

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7 February 2018

issue

9

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7 February 2018

page(s)

991-995

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7 February 2018

Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS

cites work

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https://api.crossref.org/works/10.1517%2F14728222.2013.818659

A hexanucleotide repeat expansion in C9ORF72 is the cause of chromosome 9p21-linked ALS-FTD

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728222.2013.818659

Unconventional translation of C9ORF72 GGGGCC expansion generates insoluble polypeptides specific to c9FTD/ALS

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728222.2013.818659

The C9orf72 GGGGCC repeat is translated into aggregating dipeptide-repeat proteins in FTLD/ALS.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14728222.2013.818659

RNA structure of trinucleotide repeats associated with human neurological diseases

21 January 2018

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Foci of trinucleotide repeat transcripts in nuclei of myotonic dystrophy cells and tissues

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728222.2013.818659

The structural basis of myotonic dystrophy from the crystal structure of CUG repeats

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728222.2013.818659

Expanded CUG repeats trigger aberrant splicing of ClC-1 chloride channel pre-mRNA and hyperexcitability of skeletal muscle in myotonic dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728222.2013.818659

MBNL1 is the primary determinant of focus formation and aberrant insulin receptor splicing in DM1

21 January 2018

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https://api.crossref.org/works/10.1517%2F14728222.2013.818659

Non-ATG-initiated translation directed by microsatellite expansions

21 January 2018

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https://api.crossref.org/works/10.1517%2F14728222.2013.818659

The mechanism of eukaryotic translation initiation and principles of its regulation

21 January 2018

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CGG repeat-associated translation mediates neurodegeneration in fragile X tremor ataxia syndrome

21 January 2018

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https://api.crossref.org/works/10.1517%2F14728222.2013.818659

Expanded GGGGCC repeat RNA associated with amyotrophic lateral sclerosis and frontotemporal dementia causes neurodegeneration

21 January 2018

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https://api.crossref.org/works/10.1517%2F14728222.2013.818659

The disease-associated r(GGGGCC)n repeat from the C9orf72 gene forms tract length-dependent uni- and multimolecular RNA G-quadruplex structures

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728222.2013.818659

hnRNP A3 binds to GGGGCC repeats and is a constituent of p62-positive/TDP43-negative inclusions in the hippocampus of patients with C9orf72 mutations

21 January 2018

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Mutations in prion-like domains in hnRNPA2B1 and hnRNPA1 cause multisystem proteinopathy and ALS

21 January 2018

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Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA.

21 January 2018

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From dynamic combinatorial 'hit' to lead: in vitro and in vivo activity of compounds targeting the pathogenic RNAs that cause myotonic dystrophy

21 January 2018

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Rationally designed small molecules targeting the RNA that causes myotonic dystrophy type 1 are potently bioactive

21 January 2018

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

21 January 2018

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RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1

21 January 2018

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RNA interference targeting CUG repeats in a mouse model of myotonic dystrophy

21 January 2018

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https://api.crossref.org/works/10.1517%2F14728222.2013.818659

C9orf72 hexanucleotide repeat associated with amyotrophic lateral sclerosis and frontotemporal dementia forms RNA G-quadruplexes

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14728222.2013.818659

A C9orf72 promoter repeat expansion in a Flanders-Belgian cohort with disorders of the frontotemporal lobar degeneration-amyotrophic lateral sclerosis spectrum: a gene identification study.

21 January 2018

1 reference

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10.1517/14728222.2013.818659

21 January 2018

Identifiers

DOI

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7 February 2018

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7 February 2018