Technology insight: therapy for Duchenne muscular dystrophy-an opportunity for personalized medicine? (Q37082470)

18 August 2017

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title

Technology insight: therapy for Duchenne muscular dystrophy-an opportunity for personalized medicine? (English)

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18 August 2017

personalized medicine

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2

Thomas A Rando

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18 August 2017

Leland E Lim

1

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18 August 2017

12 February 2008

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Nature Clinical Practice Neurology

18 August 2017

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18 August 2017

4

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18 August 2017

3

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18 August 2017

149-158

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Absent pituitary gland in two brothers with an oral-facial-digital syndrome resembling OFDS II and VI: a new type of OFDS?

18 August 2017

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

Experience and strategy for the molecular testing of Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

Negamycin restores dystrophin expression in skeletal and cardiac muscles of mdx mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

Prospective study of X-linked progressive muscular dystrophy in Campania

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

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

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Membrane organization of the dystrophin-glycoprotein complex

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Molecular basis of muscular dystrophies.

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

The dystrophin-glycoprotein complex, cellular signaling, and the regulation of cell survival in the muscular dystrophies

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Preferential deletion of exons in Duchenne and Becker muscular dystrophies

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Searching for the 1 in 2,400,000: a review of dystrophin gene point mutations

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Spectrum of small mutations in the dystrophin coding region

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Gene therapy strategies for Duchenne muscular dystrophy utilizing recombinant adeno-associated virus vectors.

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury.

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Dystrophin expression improves myofiber survival in mdx muscle following intramuscular plasmid DNA injection.

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Complete repair of dystrophic skeletal muscle by mesoangioblasts with enhanced migration ability

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

[About a phase I gene therapy clinical trial with a full-length dystrophin gene-plasmid in Duchenne/Becker muscular dystrophy].

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Gene therapy progress and prospects: Duchenne muscular dystrophy

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Phenotypic suppression of nonsense mutants in yeast by aminoglycoside antibiotics

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Phenotypic suppression and misreading in Saccharomyces cerevisiae

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Aminoglycoside suppression at UAG, UAA and UGA codons in Escherichia coli and human tissue culture cells

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Aminoglycoside antibiotics restore CFTR function by overcoming premature stop mutations

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Gentamicin fails to increase dystrophin expression in dystrophin-deficient muscle

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Sequence specificity of aminoglycoside-induced stop condon readthrough: potential implications for treatment of Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Premature stop codons involved in muscular dystrophies show a broad spectrum of readthrough efficiencies in response to gentamicin treatment.

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Readthrough of dystrophin stop codon mutations induced by aminoglycosides

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

PTC124 targets genetic disorders caused by nonsense mutations

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Targeted gene correction: a new strategy for molecular medicine

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Targeted gene correction by small single-stranded oligonucleotides in mammalian cells

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

A plausible mechanism for gene correction by chimeric oligonucleotides

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Mechanisms underlying targeted gene correction using chimeric RNA/DNA and single-stranded DNA oligonucleotides

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Targeted single-base correction by RNA-DNA oligonucleotides

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Correction of the mutation responsible for sickle cell anemia by an RNA-DNA oligonucleotide

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Reduction of gene repair by selenomethionine with the use of single-stranded oligonucleotides

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Strand bias in oligonucleotide-mediated dystrophin gene editing

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Involvement of ERCC1/XPF and XPG in oligodeoxynucleotide-directed gene modification

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Dynamics of nuclei of muscle fibers and connective tissue cells in normal and denervated rat muscles.

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Continuous myofiber remodeling in uninjured extraocular myofibers: myonuclear turnover and evidence for apoptosis.

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Antisense-mediated redirection of mRNA splicing

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Induced dystrophin exon skipping in human muscle explants

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Induction of revertant fibres in the mdx mouse using antisense oligonucleotides

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle.

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Poly(ethylene imine)-poly(ethylene glycol) copolymers facilitate efficient delivery of antisense oligonucleotides to nuclei of mature muscle cells of mdx mice

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Induction of dystrophin expression by exon skipping in mdx mice following intramuscular injection of antisense oligonucleotides complexed with PEG-PEI copolymers

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Intravenous infusion of an antisense oligonucleotide results in exon skipping in muscle dystrophin mRNA of Duchenne muscular dystrophy

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Local dystrophin restoration with antisense oligonucleotide PRO051

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

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

https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Dystrophin gene transcripts skipping the mdx mutation

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion

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https://api.crossref.org/works/10.1038%2FNCPNEURO0737

7 January 2021

Identifiers

DOI

10.1038/NCPNEURO0737

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18 August 2017

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PubMed ID

18268530