A dystrophic Duchenne mouse model for testing human antisense oligonucleotides. (Q52688621)

http://europepmc.org/abstract/PMC/5821388

5 May 2018

title

A dystrophic Duchenne mouse model for testing human antisense oligonucleotides. (English)

1 reference

http://europepmc.org/abstract/PMC/5821388

5 May 2018

author

Annemieke Aartsma-Rus

11

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

Maaike van Putten

12

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

6

Cor Breukel

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

10

Sjef Verbeek

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

7

Jos van der Kaa

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

4

Jill Claassens

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

5

Conny Brouwers

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

author name string

Marcel Veltrop

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

Laura van Vliet

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

Margriet Hulsker

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

Margot M Linssen

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

Johan T den Dunnen

9

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

language of work or name

English

0 references

publication date

21 February 2018

1 reference

http://europepmc.org/abstract/PMC/5821388

5 May 2018

published in

PLOS One

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

volume

13

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

issue

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

page(s)

e0193289

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

Creative Commons Attribution 4.0 International

4 June 2020

copyright license

start time

21 February 2018

1 reference

April 2022 Public Data File from Crossref

copyright status

copyrighted

0 references

cites work

Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Eteplirsen: First Global Approval

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Genome editing. The new frontier of genome engineering with CRISPR-Cas9

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Understanding the process of fibrosis in Duchenne muscular dystrophy.

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Assessing functional performance in the mdx mouse model

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Generation of embryonic stem cells and mice for duchenne research

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Newborn bloodspot screening for Duchenne muscular dystrophy: 21 years experience in Wales (UK).

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

TAL Effector-Nucleotide Targeter (TALE-NT) 2.0: tools for TAL effector design and target prediction

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

The effects of low levels of dystrophin on mouse muscle function and pathology

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Systemic administration of PRO051 in Duchenne's muscular dystrophy

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Targeting fibrosis in Duchenne muscular dystrophy

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Accurate quantification of dystrophin mRNA and exon skipping levels in duchenne muscular dystrophy.

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Improved embryonic stem cell technologies.

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Compositional analysis of muscle in boys with Duchenne muscular dystrophy using MR imaging.

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides.

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

X chromosome-linked muscular dystrophy (mdx) in the mouse

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Exploring the molecular basis for variability among patients with Becker muscular dystrophy: dystrophin gene and protein studies

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Accurate Dystrophin Quantification in Mouse Tissue; Identification of New and Evaluation of Existing Methods

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Genome Editing in Mice Using TALE Nucleases

13 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Clinical characterisation of Becker muscular dystrophy patients predicts favourable outcome in exon-skipping therapy.

13 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

13 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.

13 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Targeted Disruption of Exon 52 in the Mouse Dystrophin Gene Induced Muscle Degeneration Similar to That Observed in Duchenne Muscular Dystrophy

13 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Comparison of skeletal muscle pathology and motor function of dystrophin and utrophin deficient mouse strains

13 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Generation and Characterization of Transgenic Mice with the Full-length HumanDMDGene

30 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5821388

Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne's or Becker's muscular dystrophy

30 November 2018

1 reference

12 December 2020

Three mouse models of muscular dystrophy: the natural history of strength and fatigue in dystrophin-, dystrophin/utrophin-, and laminin α2-deficient mice

1 reference

12 December 2020

The muscular dystrophies

1 reference

12 December 2020

Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy

1 reference

12 December 2020

FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides

1 reference

12 December 2020

10.1371/JOURNAL.PONE.0193289

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29466448 AND SRC:MED&resulttype=core&format=json

4 June 2020

PubMed publication ID

29466448

1 reference