Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy (Q33678893)

28 July 2017

title

Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy (English)

1 reference

Duchenne muscular dystrophy

28 July 2017

1 reference

1 reference

Jane T Seto

1

1 reference

28 July 2017

Niclas E Bengtsson

series ordinal

2

1 reference

28 July 2017

Jeffrey S Chamberlain

series ordinal

3

1 reference

28 July 2017

publication date

1 June 2014

1 reference

Current pediatrics reports

28 July 2017

published in

1 reference

28 July 2017

volume

2

1 reference

28 July 2017

issue

2

1 reference

28 July 2017

page(s)

102-112

1 reference

Orphan drug development in muscular dystrophy: update on two large clinical trials of dystrophin rescue therapies

28 July 2017

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Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy

21 June 2018

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21 June 2018

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21 June 2018

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21 June 2018

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Update on the treatment of Duchenne muscular dystrophy

21 June 2018

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Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy

21 June 2018

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

21 June 2018

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21 June 2018

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21 June 2018

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Human ES- and iPS-derived myogenic progenitors restore DYSTROPHIN and improve contractility upon transplantation in dystrophic mice.

21 June 2018

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Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors

21 June 2018

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Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping

21 June 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy

21 June 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector

21 June 2018

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Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma

21 June 2018

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21 June 2018

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Transplanted myoblasts can migrate several millimeters to fuse with damaged myofibers in nonhuman primate skeletal muscle

21 June 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Stem cell-mediated transfer of a human artificial chromosome ameliorates muscular dystrophy.

21 June 2018

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An absolute requirement for Pax7-positive satellite cells in acute injury-induced skeletal muscle regeneration

21 June 2018

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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

21 June 2018

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Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse

21 June 2018

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Functional myogenic engraftment from mouse iPS cells.

21 June 2018

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Systemic administration of PRO051 in Duchenne's muscular dystrophy

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Expression of human alpha1-antitrypsin in mice and dogs following AAV6 vector-mediated gene transfer to the lungs.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Follistatin gene delivery enhances muscle growth and strength in nonhuman primates

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Cell based therapy for Duchenne muscular dystrophy

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Evaluation of vascular delivery methodologies to enhance rAAV6-mediated gene transfer to canine striated musculature

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Truncated dystrophins can influence neuromuscular synapse structure.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Molecular and cellular adaptations to chronic myotendinous strain injury in mdx mice expressing a truncated dystrophin

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Protein trans-splicing as a means for viral vector-mediated in vivo gene therapy

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Recombinant adeno-associated virus transduction and integration

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Manufacturing and characterizing AAV-based vectors for use in clinical studies

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Ischemic central necrosis in pockets of transplanted myoblasts in nonhuman primates: implications for cell-transplantation strategies

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

The role of Pax genes in the development of tissues and organs: Pax3 and Pax7 regulate muscle progenitor cell functions

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

PTC124 targets genetic disorders caused by nonsense mutations

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Stem and progenitor cells in skeletal muscle development, maintenance, and therapy

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: Implications for gene therapy using AAV vectors

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

MyoD induces myogenic differentiation through cooperation of its NH2- and COOH-terminal regions.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Satellite cell of skeletal muscle fibers

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Activation of muscle-specific genes in pigment, nerve, fat, liver, and fibroblast cell lines by forced expression of MyoD.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

21 June 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Eteplirsen for the treatment of Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Dystrophin immunity in Duchenne's muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Percutaneous transendocardial delivery of self-complementary adeno-associated virus 6 in the canine

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Immune evasion by muscle-specific gene expression in dystrophic muscle

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Modulation of Starling forces and muscle fiber maturity permits adenovirus-mediated gene transfer to adult dystrophic (mdx) mice by the intravascular route

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Dystrophin expression in muscles of duchenne muscular dystrophy patients after high-density injections of normal myogenic cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Dystrophin expression in myofibers of Duchenne muscular dystrophy patients following intramuscular injections of normal myogenic cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Direct isolation of satellite cells for skeletal muscle regeneration.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Transendocardial delivery of AAV6 results in highly efficient and global cardiac gene transfer in rhesus macaques

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4036820

Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human

30 October 2018

1 reference

https://api.crossref.org/works/10.1007%2FS40124-014-0044-X

Myoblast Transfer in the Treatment of Duchenne's Muscular Dystrophy

21 January 2018

1 reference

12 December 2020

Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation

1 reference

12 December 2020

Codon and mRNA Sequence Optimization of Microdystrophin Transgenes Improves Expression and Physiological Outcome in Dystrophic mdx Mice Following AAV2/8 Gene Transfer

1 reference

12 December 2020

Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression

1 reference

12 December 2020

Local dystrophin restoration with antisense oligonucleotide PRO051

1 reference

12 December 2020

Rapid death of injected myoblasts in myoblast transfer therapy

1 reference

12 December 2020

10.1007/S40124-014-0044-X

Identifiers

DOI

1 reference

Dimensions Publication ID

28 July 2017

0 references

1 reference

28 July 2017

1 reference