Skipping Multiple Exons to Treat DMD-Promises and Challenges. (Q52429827)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29301272 AND SRC:MED&resulttype=core&format=json

8 April 2020

title

Skipping Multiple Exons to Treat DMD-Promises and Challenges (English)

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8 April 2020

3

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8 April 2020

author name string

Tejal Aslesh

series ordinal

1

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8 April 2020

Rika Maruyama

series ordinal

2

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8 April 2020

publication date

2 January 2018

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8 April 2020

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8 April 2020

volume

6

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8 April 2020

issue

1

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Creative Commons Attribution 4.0 International

8 April 2020

copyright license

start time

2 January 2018

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April 2022 Public Data File from Crossref

copyright status

copyrighted

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cites work

Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy

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Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies

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

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Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.

22 May 2018

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Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice

22 May 2018

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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

22 May 2018

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Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.

22 May 2018

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

22 May 2018

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Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?

22 May 2018

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Genetic therapeutic approaches for Duchenne muscular dystrophy

22 May 2018

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Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through

22 May 2018

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22 May 2018

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22 May 2018

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

22 May 2018

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22 May 2018

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22 May 2018

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22 May 2018

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22 May 2018

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22 May 2018

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Follow-up of three patients with a large in-frame deletion of exons 45-55 in the Duchenne muscular dystrophy (DMD) gene

22 May 2018

1 reference

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Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo.

22 May 2018

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Molecular mechanisms of muscular dystrophies: old and new players

22 May 2018

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

22 May 2018

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Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics.

22 May 2018

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Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

22 May 2018

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Dystrophin and mutations: one gene, several proteins, multiple phenotypes

22 May 2018

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DMD(mdx3Cv) and DMD(mdx4Cv) dystrophin mutations in mice: rapid polymerase chain reaction genotyping

22 May 2018

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Induction of exon skipping of the dystrophin transcript in lymphoblastoid cells by transfecting an antisense oligodeoxynucleotide complementary to an exon recognition sequence

22 May 2018

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The role of the dystrophin-glycoprotein complex in the molecular pathogenesis of muscular dystrophies.

22 May 2018

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Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals

22 May 2018

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Duchenne muscular dystrophy: deficiency of dystrophin at the muscle cell surface

22 May 2018

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The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion

22 May 2018

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Subcellular fractionation of dystrophin to the triads of skeletal muscle

22 May 2018

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Very mild muscular dystrophy associated with the deletion of 46% of dystrophin

22 May 2018

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Controversies about Duchenne muscular dystrophy. (1) Neonatal screening

22 May 2018

1 reference

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Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle

22 May 2018

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Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy.

13 August 2018

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Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach

13 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5874658

Systemic Delivery of Morpholinos to Skip Multiple Exons in a Dog Model of 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=5874658

Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of 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=5874658

Deletion of exons 3-9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon 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=5874658

The Pathology of Paralysis with Muscular Degeneration (Paralysie Myosclerotique), or Paralysis with Apparent Hypertrophy

13 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5874658

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

13 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5874658

Asymptomatic Becker muscular dystrophy in a family with a multiexon deletion

13 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5874658

CRISPR-mediated genome editing and human diseases

13 August 2018

1 reference

Crossref

https://api.crossref.org/works/10.3390/BIOMEDICINES6010001

Dystrophin expression and somatic reversion in prednisone-treated and untreated Duchenne dystrophy. CIDD Study Group

21 January 2018

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12 December 2020

Molecular and clinical correlations of deletions leading to Duchenne and Becker muscular dystrophies

1 reference

12 December 2020

Expansion of revertant fibers in dystrophic mdx muscles reflects activity of muscle precursor cells and serves as an index of muscle regeneration

1 reference

12 December 2020

Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy

1 reference

12 December 2020

Peptide-morpholino conjugate: a promising therapeutic for Duchenne muscular dystrophy

1 reference

12 December 2020

Diaphragm rescue alone prevents heart dysfunction in dystrophic mice

1 reference

12 December 2020

10.3390/BIOMEDICINES6010001

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29301272 AND SRC:MED&resulttype=core&format=json

8 April 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29301272 AND SRC:MED&resulttype=core&format=json

8 April 2020

PubMed publication ID

29301272

1 reference