(Q38210259)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24809691%20AND%20SRC:MED&resulttype=core&format=json

17 March 2020

title

Antisense oligonucleotide therapy for the treatment of C9ORF72 ALS/FTD diseases (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24809691%20AND%20SRC:MED&resulttype=core&format=json

amyotrophic lateral sclerosis

17 March 2020

main subject

1 reference

4

1 reference

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17 March 2020

Francesca Magri

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24809691%20AND%20SRC:MED&resulttype=core&format=json

17 March 2020

Nereo Bresolin

7

1 reference

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17 March 2020

Giacomo P Comi

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24809691%20AND%20SRC:MED&resulttype=core&format=json

17 March 2020

Stefania Corti

9

1 reference

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17 March 2020

author name string

Giulietta Riboldi

1

1 reference

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17 March 2020

Chiara Zanetta

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24809691%20AND%20SRC:MED&resulttype=core&format=json

17 March 2020

Michela Ranieri

3

1 reference

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17 March 2020

Chiara Simone

5

1 reference

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17 March 2020

publication date

9 May 2014

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24809691%20AND%20SRC:MED&resulttype=core&format=json

17 March 2020

published in

Molecular Neurobiology

1 reference

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17 March 2020

volume

50

1 reference

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17 March 2020

issue

3

1 reference

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17 March 2020

page(s)

721-732

1 reference

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

17 March 2020

cites work

1 reference

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

21 January 2018

1 reference

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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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Lack of C9ORF72 coding mutations supports a gain of function for repeat expansions in amyotrophic lateral sclerosis

21 January 2018

1 reference

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C9orf72 G4C2 repeat expansions in Alzheimer's disease and mild cognitive impairment.

21 January 2018

1 reference

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C9orf72 expansions are the most common genetic cause of Huntington disease phenocopies

21 January 2018

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TAR DNA-binding protein 43 in neurodegenerative disease

21 January 2018

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Deciphering amyotrophic lateral sclerosis: what phenotype, neuropathology and genetics are telling us about pathogenesis

21 January 2018

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FTD and ALS: a tale of two diseases

21 January 2018

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Converging mechanisms in ALS and FTD: disrupted RNA and protein homeostasis

21 January 2018

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Exome sequencing reveals VCP mutations as a cause of familial ALS

21 January 2018

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The genetics and neuropathology of amyotrophic lateral sclerosis

21 January 2018

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Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis

21 January 2018

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Recent advances in the genetics of the ALS-FTLD complex

21 January 2018

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Repair of pre-mRNA splicing: prospects for a therapy for spinal muscular atrophy.

21 January 2018

1 reference

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Antisense oligonucleotides for the treatment of spinal muscular atrophy

21 January 2018

1 reference

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SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy

21 January 2018

1 reference

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Antisense oligonucleotide therapy for neurodegenerative disease.

21 January 2018

1 reference

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An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

21 January 2018

1 reference

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RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention

21 January 2018

1 reference

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Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

21 January 2018

1 reference

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Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion

21 January 2018

1 reference

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Frequency of the C9orf72 hexanucleotide repeat expansion in patients with amyotrophic lateral sclerosis and frontotemporal dementia: a cross-sectional study

21 January 2018

1 reference

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Microsatellite repeat instability and neurological disease

21 January 2018

1 reference

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Repeat instability: mechanisms of dynamic mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS12035-014-8724-7

Cellular toxicity of expanded RNA repeats: focus on RNA foci

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS12035-014-8724-7

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS12035-014-8724-7

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS12035-014-8724-7

Hypermethylation of the CpG island near the G4C2 repeat in ALS with a C9orf72 expansion

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS12035-014-8724-7

Homozygosity for the C9orf72 GGGGCC repeat expansion in frontotemporal dementia.

21 January 2018

1 reference

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C9orf72 hexanucleotide repeat expansions in clinical Alzheimer disease

21 January 2018

1 reference

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS12035-014-8724-7

Newly identified ADAR-mediated A-to-I editing positions as a tool for ALS research

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS12035-014-8724-7

Determination of editors at the novel A-to-I editing positions

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS12035-014-8724-7

Profound downregulation of the RNA editing enzyme ADAR2 in ALS spinal motor neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS12035-014-8724-7

Late-onset motoneuron disease caused by a functionally modified AMPA receptor subunit

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS12035-014-8724-7

Glutamate receptors: RNA editing and death of motor neurons

21 January 2018

1 reference

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Modeling key pathological features of frontotemporal dementia with C9ORF72 repeat expansion in iPSC-derived human neurons

21 January 2018

1 reference

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RNA-binding proteins in microsatellite expansion disorders: mediators of RNA toxicity

21 January 2018

1 reference

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RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform

21 January 2018

1 reference

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Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy

21 January 2018

1 reference

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

21 January 2018

1 reference

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Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles

21 January 2018

1 reference

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Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model

21 January 2018

1 reference

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Splicing therapy for neuromuscular disease

21 January 2018

1 reference

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Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: removal of the CAG containing exon

21 January 2018

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Antisense oligonucleotides: rising stars in eliminating RNA toxicity in myotonic dystrophy.

21 January 2018

1 reference

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Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy.

21 January 2018

1 reference

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Design and analysis of effects of triplet repeat oligonucleotides in cell models for myotonic dystrophy

21 January 2018

1 reference

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Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.

21 January 2018

1 reference

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Exon skipping quantification by quantitative reverse-transcription polymerase chain reaction in Duchenne muscular dystrophy patients treated with the antisense oligomer eteplirsen

21 January 2018

1 reference

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Systemic administration of PRO051 in Duchenne's muscular dystrophy

21 January 2018

1 reference

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Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models

21 January 2018

1 reference

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The mouse C9ORF72 ortholog is enriched in neurons known to degenerate in ALS and FTD.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS12035-014-8724-7

Antisense-based therapy for the treatment of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS12035-014-8724-7

Analysis of hnRNPA1, A2/B1, and A3 genes in patients with amyotrophic lateral sclerosis.

21 January 2018

1 reference

12 December 2020

C9ORF72 intermediate repeat copies are a significant risk factor for Parkinson disease

1 reference

12 December 2020

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

1 reference

12 December 2020

Antisense oligonucleotides: basic concepts and mechanisms

1 reference

12 December 2020

RAN proteins and RNA foci from antisense transcripts in C9ORF72 ALS and frontotemporal dementia

1 reference

12 December 2020

RNA-mediated neurodegeneration in repeat expansion disorders

1 reference

12 December 2020

Loss of function of C9orf72 causes motor deficits in a zebrafish model of amyotrophic lateral sclerosis.

1 reference

12 December 2020