(Q64947331)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29947794%20AND%20SRC:MED&resulttype=core&format=json

2 April 2020

title

Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells (English)

1 reference

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2 April 2020

1 reference

1 reference

0 references

5

Nisha Nair

1 reference

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Mattia Francesco Maria Gerli

2 April 2020

9

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2 April 2020

Arnaud F Klein

10

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2 April 2020

Peter In'T Veld

17

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2 April 2020

Denis Furling

18

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Francesco Saverio Tedesco

2 April 2020

19

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2 April 2020

Joris R Vermeesch

20

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2 April 2020

Thierry VandenDriessche

23

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2 April 2020

21

J Keith Joung

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2 April 2020

author name string

Sumitava Dastidar

1

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2 April 2020

Simon Ardui

2

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2 April 2020

Kshitiz Singh

3

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2 April 2020

Debanjana Majumdar

4

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2 April 2020

Yanfang Fu

6

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2 April 2020

Deepak Reyon

7

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2 April 2020

Ermira Samara

8

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2 April 2020

Wito De Schrijver

11

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2 April 2020

Jaitip Tipanee

12

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2 April 2020

Sara Seneca

13

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2 April 2020

Warut Tulalamba

14

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2 April 2020

Hui Wang

15

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2 April 2020

Yoke Chin Chai

16

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2 April 2020

Marinee K Chuah

22

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2 April 2020

language of work or name

English

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publication date

1 September 2018

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2 April 2020

published in

Nucleic Acids Research

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2 April 2020

volume

46

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2 April 2020

issue

16

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2 April 2020

page(s)

8275-8298

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Creative Commons Attribution-NonCommercial 4.0 International

2 April 2020

copyright license

start time

27 June 2018

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April 2022 Public Data File from Crossref

copyright status

copyrighted

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describes a project that uses

ImageJ

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11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC6144820/fullTextXML

inferred from PubMed Central ID database lookup

cites work

Myotonic dystrophy mutation: an unstable CTG repeat in the 3' untranslated region of the gene

1 reference

12 December 2020

An unstable triplet repeat in a gene related to myotonic muscular dystrophy

1 reference

12 December 2020

Normal variation at the myotonic dystrophy locus in global human populations

1 reference

12 December 2020

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

1 reference

12 December 2020

Myotonic dystrophy: size- and sex-dependent dynamics of CTG meiotic instability, and somatic mosaicism.

1 reference

12 December 2020

Structure and genomic sequence of the myotonic dystrophy (DM kinase) gene

1 reference

12 December 2020

Localization of trinucleotide repeat sequences in myotonic dystrophy cells using a single fluorochrome-labeled PNA probe

1 reference

12 December 2020

Ribozyme-mediated trans-splicing of a trinucleotide repeat.

1 reference

12 December 2020

Myotonic dystrophy in transgenic mice expressing an expanded CUG repeat

1 reference

12 December 2020

Mouse tissue culture models of unstable triplet repeats: in vitro selection for larger alleles, mutational expansion bias and tissue specificity, but no association with cell division rates

1 reference

12 December 2020

Mice transgenic for the human myotonic dystrophy region with expanded CTG repeats display muscular and brain abnormalities.

1 reference

12 December 2020

Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo

1 reference

12 December 2020

Mesoangioblasts--vascular progenitors for extravascular mesodermal tissues

1 reference

12 December 2020

A muscleblind knockout model for myotonic dystrophy

1 reference

12 December 2020

RNA pathogenesis of the myotonic dystrophies

1 reference

12 December 2020

Cytoplasmic and nuclear retained DMPK mRNAs are targets for RNA interference in myotonic dystrophy cells.

1 reference

12 December 2020

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

1 reference

12 December 2020

Altered mRNA splicing of the skeletal muscle ryanodine receptor and sarcoplasmic/endoplasmic reticulum Ca2+-ATPase in myotonic dystrophy type 1.

1 reference

12 December 2020

Failure of MBNL1-dependent post-natal splicing transitions in myotonic dystrophy

1 reference

12 December 2020

Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells

1 reference

12 December 2020

Elevation of RNA-binding protein CUGBP1 is an early event in an inducible heart-specific mouse model of myotonic dystrophy

1 reference

12 December 2020

Defective mRNA in myotonic dystrophy accumulates at the periphery of nuclear splicing speckles

1 reference

12 December 2020

Induction of pluripotent stem cells from adult human fibroblasts by defined factors

1 reference

12 December 2020

CTG repeat instability in a human embryonic stem cell line carrying the myotonic dystrophy type 1 mutation.

1 reference

12 December 2020

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

1 reference

12 December 2020

Virus-free induction of pluripotency and subsequent excision of reprogramming factors

1 reference

12 December 2020

Zinc-finger directed double-strand breaks within CAG repeat tracts promote repeat instability in human cells

1 reference

12 December 2020

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

1 reference

12 December 2020

Aberrant alternative splicing and extracellular matrix gene expression in mouse models of myotonic dystrophy.

1 reference

12 December 2020

A nonviral minicircle vector for deriving human iPS cells

1 reference

12 December 2020

Molecular therapy in myotonic dystrophy: focus on RNA gain-of-function

1 reference

12 December 2020

Huntington's and myotonic dystrophy hESCs: down-regulated trinucleotide repeat instability and mismatch repair machinery expression upon differentiation

1 reference

12 December 2020

The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA

1 reference

12 December 2020

Stem-cell gene therapy for the Wiskott-Aldrich syndrome

1 reference

12 December 2020

Selective silencing of mutated mRNAs in DM1 by using modified hU7-snRNAs

1 reference

12 December 2020

Survival and CTG repeat expansion in adults with myotonic dystrophy type 1

1 reference

12 December 2020

Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

1 reference

12 December 2020

Misregulated alternative splicing of BIN1 is associated with T tubule alterations and muscle weakness in myotonic dystrophy.

1 reference

12 December 2020

RNA steady-state defects in myotonic dystrophy are linked to nuclear exclusion of SHARP.

1 reference

12 December 2020

Cellular toxicity of expanded RNA repeats: focus on RNA foci

1 reference

12 December 2020

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

1 reference

12 December 2020

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

1 reference

12 December 2020

The RNA-binding protein Staufen1 is increased in DM1 skeletal muscle and promotes alternative pre-mRNA splicing

1 reference

12 December 2020

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

1 reference

12 December 2020

Transplantation of genetically corrected human iPSC-derived progenitors in mice with limb-girdle muscular dystrophy.

1 reference

12 December 2020

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

1 reference

12 December 2020

Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria

1 reference

12 December 2020

The myotonic dystrophies: molecular, clinical, and therapeutic challenges

1 reference

12 December 2020

Sequencing the unsequenceable: expanded CGG-repeat alleles of the fragile X gene

1 reference

12 December 2020

Assessing telomeric DNA content in pediatric cancers using whole-genome sequencing data

1 reference

12 December 2020

Multiplex genome engineering using CRISPR/Cas systems

1 reference

12 December 2020

RNA-guided human genome engineering via Cas9

1 reference

12 December 2020

RNA-programmed genome editing in human cells

1 reference

12 December 2020

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

1 reference

12 December 2020

Molecular genetics and genetic testing in myotonic dystrophy type 1.

1 reference

12 December 2020

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

1 reference

12 December 2020

CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity

1 reference

12 December 2020

High-content screening identifies small molecules that remove nuclear foci, affect MBNL distribution and CELF1 protein levels via a PKC-independent pathway in myotonic dystrophy cell lines

1 reference

12 December 2020

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

1 reference

12 December 2020

Tandem repeat modification during double-strand break repair induced by an engineered TAL effector nuclease in zebrafish genome

1 reference

12 December 2020

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

1 reference

12 December 2020

Highly specific contractions of a single CAG/CTG trinucleotide repeat by TALEN in yeast.

1 reference

12 December 2020

Polyglutamine Disease Modeling: Epitope Based Screen for Homologous Recombination using CRISPR/Cas9 System

1 reference

12 December 2020

Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects

1 reference

12 December 2020

Disease-associated CAG·CTG triplet repeats expand rapidly in non-dividing mouse cells, but cell cycle arrest is insufficient to drive expansion

1 reference

12 December 2020

Targeted genome editing in human repopulating haematopoietic stem cells

1 reference

12 December 2020

Engineering human tumour-associated chromosomal translocations with the RNA-guided CRISPR-Cas9 system

1 reference

12 December 2020

In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system.

1 reference

12 December 2020

Simple and rapid in vivo generation of chromosomal rearrangements using CRISPR/Cas9 technology

1 reference

12 December 2020

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites

1 reference

12 December 2020

Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases.

1 reference

12 December 2020

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

1 reference

12 December 2020

Prevalence of muscular dystrophies: a systematic literature review

1 reference

12 December 2020

Unbiased detection of off-target cleavage by CRISPR-Cas9 and TALENs using integrase-defective lentiviral vectors.

1 reference

12 December 2020

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

1 reference

12 December 2020

Genome modification leads to phenotype reversal in human myotonic dystrophy type 1 induced pluripotent stem cell-derived neural stem cells

1 reference

12 December 2020

Loss of muscleblind-like 1 results in cardiac pathology and persistence of embryonic splice isoforms

1 reference

12 December 2020

In vivo genome editing using Staphylococcus aureus Cas9

1 reference

12 December 2020

Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells.

1 reference

12 December 2020

Pluripotent Stem Cells for Gene Therapy of Degenerative Muscle Diseases.

1 reference

12 December 2020

Therapeutic Approaches for Dominant Muscle Diseases: Highlight on Myotonic Dystrophy

1 reference

12 December 2020

Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9.

1 reference

12 December 2020

SMRT Sequencing of Long Tandem Nucleotide Repeats in SCA10 Reveals Unique Insight of Repeat Expansion Structure

1 reference

12 December 2020

Simple Repeat-Primed PCR Analysis of the Myotonic Dystrophy Type 1 Gene in a Clinical Diagnostics Environment.

1 reference

12 December 2020

Reversion of FMR1 Methylation and Silencing by Editing the Triplet Repeats in Fragile X iPSC-Derived Neurons.

1 reference

12 December 2020

Rationally engineered Cas9 nucleases with improved specificity

1 reference

12 December 2020

piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts

1 reference

12 December 2020

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.

1 reference

12 December 2020

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

1 reference

12 December 2020

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

1 reference

12 December 2020

High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects.

1 reference

12 December 2020

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

1 reference

12 December 2020

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

1 reference

12 December 2020

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

1 reference

12 December 2020

A decade of transcription factor-mediated reprogramming to pluripotency

1 reference

12 December 2020

CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy

1 reference

12 December 2020

CRISPR/Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse

1 reference

12 December 2020

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

1 reference

12 December 2020

Quantification of telomere features in tumor tissue sections by an automated 3D imaging-based workflow

1 reference

12 December 2020

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

1 reference

12 December 2020

Detecting AGG Interruptions in Male and Female FMR1 Premutation Carriers by Single-Molecule Sequencing

1 reference

12 December 2020

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

1 reference

12 December 2020

Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9.

1 reference

12 December 2020

CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR-Cas9 nuclease off-targets.

1 reference

12 December 2020

Unexpected mutations after CRISPR–Cas9 editing in vivo

1 reference

12 December 2020

CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease

1 reference

12 December 2020

CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients

1 reference

12 December 2020

Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9

1 reference

12 December 2020

Identifiers

DOI

10.1093/NAR/GKY548

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29947794%20AND%20SRC:MED&resulttype=core&format=json

2 April 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29947794%20AND%20SRC:MED&resulttype=core&format=json

2 April 2020

1 reference