Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9. (Q38695021)

http://europepmc.org/abstract/PMC/5873302

2 July 2018

title

Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9. (English)

1 reference

http://europepmc.org/abstract/PMC/5873302

2 July 2018

1 reference

0 references

4

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28803727%20AND%20SRC:MED&resulttype=core&format=json

9 June 2020

Isaac A Chaim

7

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9 June 2020

Stefan Aigner

11

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9 June 2020

Kevin D Corbett

14

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9 June 2020

Maurice S. Swanson

15

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9 June 2020

Gene W Yeo

16

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9 June 2020

author name string

Ranjan Batra

1

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9 June 2020

David A Nelles

2

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9 June 2020

Elaine Pirie

3

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9 June 2020

Ryan J Marina

5

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9 June 2020

Harrison Wang

6

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9 June 2020

James D Thomas

8

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9 June 2020

Nigel Zhang

9

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9 June 2020

Vu Nguyen

10

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9 June 2020

Sebastian Markmiller

12

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9 June 2020

Guangbin Xia

13

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9 June 2020

language of work or name

English

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publication date

10 August 2017

1 reference

http://europepmc.org/abstract/PMC/5873302

2 July 2018

published in

Cell

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28803727%20AND%20SRC:MED&resulttype=core&format=json

9 June 2020

volume

170

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9 June 2020

page(s)

899-912.e10

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9 June 2020

issue

5

1 reference

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Unexpected mutations after CRISPR–Cas9 editing in vivo

9 June 2020

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Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice

3 July 2018

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3 July 2018

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RNA-binding protein CPEB1 remodels host and viral RNA landscapes

3 July 2018

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Genome Therapy of Myotonic Dystrophy Type 1 iPS Cells for Development of Autologous Stem Cell Therapy

3 July 2018

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Programmable RNA Tracking in Live Cells with CRISPR/Cas9.

3 July 2018

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RAN Translation in Huntington Disease

3 July 2018

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Conformational control of DNA target cleavage by CRISPR-Cas9.

3 July 2018

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Gene therapy returns to centre stage

3 July 2018

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The C9orf72 repeat expansion disrupts nucleocytoplasmic transport.

3 July 2018

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Distinct brain transcriptome profiles in C9orf72-associated and sporadic ALS.

3 July 2018

1 reference

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C9ORF72 GGGGCC Expanded Repeats Produce Splicing Dysregulation which Correlates with Disease Severity in Amyotrophic Lateral Sclerosis

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Global insights into alternative polyadenylation regulation

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

3 July 2018

1 reference

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RNA toxicity and missplicing in the common eye disease fuchs endothelial corneal dystrophy.

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Programmable RNA recognition and cleavage by CRISPR/Cas9.

3 July 2018

1 reference

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Loss of MBNL leads to disruption of developmentally regulated alternative polyadenylation in RNA-mediated disease.

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

RNA-binding protein misregulation in microsatellite expansion disorders

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Improved vectors and genome-wide libraries for CRISPR screening

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

DNA interrogation by the CRISPR RNA-guided endonuclease Cas9.

3 July 2018

1 reference

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Dynamic imaging of genomic loci in living human cells by an optimized CRISPR/Cas system

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Compound loss of muscleblind-like function in myotonic dystrophy

3 July 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

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

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Treatment of type 1 myotonic dystrophy by engineering site-specific RNA endonucleases that target (CUG)(n) repeats

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

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

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Splicing biomarkers of disease severity in myotonic dystrophy

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

OLego: fast and sensitive mapping of spliced mRNA-Seq reads using small seeds.

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Generation of neural cells from DM1 induced pluripotent stem cells as cellular model for the study of central nervous system neuropathogenesis

3 July 2018

1 reference

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

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

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

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Muscleblind-like 2-mediated alternative splicing in the developing brain and dysregulation in myotonic dystrophy

3 July 2018

1 reference

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Fiji: an open-source platform for biological-image analysis

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

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

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

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

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

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

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Cellular toxicity of expanded RNA repeats: focus on RNA foci

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

An antisense CAG repeat transcript at JPH3 locus mediates expanded polyglutamine protein toxicity in Huntington's disease-like 2 mice

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Developments in RNA splicing and disease

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Partners in crime: bidirectional transcription in unstable microsatellite disease

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Repeat expansion disease: progress and puzzles in disease pathogenesis

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

The myotonic dystrophies: diagnosis and management

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

RNA gain-of-function in spinocerebellar ataxia type 8.

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

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

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Mechanisms of RNA-mediated disease.

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Crystal structure of the PIN domain of human telomerase-associated protein EST1A

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophy

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Lentivirus-delivered stable gene silencing by RNAi in primary cells

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Reversible cross-linking combined with immunoprecipitation to study RNA-protein interactions in vivo

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Molecular basis for impaired muscle differentiation in myotonic dystrophy

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Myotonic dystrophy type 2 caused by a CCTG expansion in intron 1 of ZNF9

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Polyglutamine disease and neuronal cell death.

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Large expansion of the ATTCT pentanucleotide repeat in spinocerebellar ataxia type 10.

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes.

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Androgen receptor gene mutations in X-linked spinal and bulbar muscular atrophy

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Fragile X genotype characterized by an unstable region of DNA.

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Instability of a 550-base pair DNA segment and abnormal methylation in fragile X syndrome

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Genetic variation at five trimeric and tetrameric tandem repeat loci in four human population groups

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Identification of a gene (FMR-1) containing a CGG repeat coincident with a breakpoint cluster region exhibiting length variation in fragile X syndrome

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

CRISPR/Cas9-Induced (CTG⋅CAG)n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing.

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

Expansion of a novel CAG trinucleotide repeat in the 5' region of PPP2R2B is associated with SCA12.

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5873302

10.1016/J.CELL.2017.07.010

31 August 2018

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28803727%20AND%20SRC:MED&resulttype=core&format=json

9 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28803727%20AND%20SRC:MED&resulttype=core&format=json

9 June 2020

1 reference