Molecular pathophysiology and targeted therapeutics for muscular dystrophy (Q34360418)

1 August 2017

title

Molecular pathophysiology and targeted therapeutics for muscular dystrophy (English)

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1 August 2017

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Hoffman EP

1

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1 August 2017

Dressman D

series ordinal

2

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1 August 2017

publication date

1 September 2001

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Trends in Pharmacological Sciences

1 August 2017

published in

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1 August 2017

volume

22

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1 August 2017

issue

9

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1 August 2017

page(s)

465-470

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Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy

1 August 2017

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Studies of sarcolemmal integrity in myopathic muscle

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7 January 2021

Animal models for muscular dystrophy show different patterns of sarcolemmal disruption.

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Alteration in calcium handling at the subcellular level in mdx myotubes.

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Expression profiling in the muscular dystrophies: identification of novel aspects of molecular pathophysiology

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Contrast agent-enhanced magnetic resonance imaging of skeletal muscle damage in animal models of muscular dystrophy

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Functional muscle ischemia in neuronal nitric oxide synthase-deficient skeletal muscle of children with Duchenne muscular dystrophy

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The serum nitric oxide levels in patients with Duchenne muscular dystrophy

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Impaired nitrergic relaxations in the gastric fundus of dystrophic (mdx) mice

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Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice

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

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Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for duchenne muscular dystrophy

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

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

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

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7 January 2021

Adeno-associated virus in normal and myositis human skeletal muscle

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Selective uptake and sustained expression of AAV vectors following subcutaneous delivery

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7 January 2021

Adeno-associated virus site-specifically integrates into a muscle-specific DNA region

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7 January 2021

rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy

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

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Rescue of skeletal muscles of gamma-sarcoglycan-deficient mice with adeno-associated virus-mediated gene transfer.

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Full functional rescue of a complete muscle (TA) in dystrophic hamsters by adeno-associated virus vector-directed gene therapy

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7 January 2021

Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization

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7 January 2021

Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

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7 January 2021

Hyaluronidase enhances recombinant adeno-associated virus (rAAV)-mediated gene transfer in the rat skeletal muscle.

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7 January 2021

Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors

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7 January 2021

Immune response to adenovirus-delivered antigens upregulates utrophin and results in mitigation of muscle pathology in mdx mice

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7 January 2021

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

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7 January 2021

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

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7 January 2021

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

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7 January 2021

Is myoblast transplantation effective?

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7 January 2021

Clonal isolation of muscle-derived cells capable of enhancing muscle regeneration and bone healing

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7 January 2021

Expression of CD34 and Myf5 defines the majority of quiescent adult skeletal muscle satellite cells

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7 January 2021

Transplacental injection of somite-derived cells in mdx mouse embryos for the correction of dystrophin deficiency

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7 January 2021

Thymic myoid cells as a source of cells for myoblast transfer

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7 January 2021

Why do cultured transplanted myoblasts die in vivo? DNA quantification shows enhanced survival of donor male myoblasts in host mice depleted of CD4+ and CD8+ cells or Nk1.1+ cells

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7 January 2021

A factor implicated in the myogenic conversion of nonmuscle cells derived from the mouse dermis

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7 January 2021

Regenerative capacity and the number of satellite cells in soleus muscles of normal and mdx mice

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7 January 2021

Increased expression of IGF-binding protein-5 in Duchenne muscular dystrophy (DMD) fibroblasts correlates with the fibroblast-induced downregulation of DMD myoblast growth: an in vitro analysis

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7 January 2021

Muscle regeneration by bone marrow-derived myogenic progenitors

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7 January 2021

Dystrophin expression in the mdx mouse restored by stem cell transplantation

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7 January 2021

A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy

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7 January 2021

T-cell-dependent fibrosis in the mdx dystrophic mouse

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7 January 2021

Altered pathological progression of diaphragm and quadriceps muscle in TNF-deficient, dystrophin-deficient mice

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7 January 2021

Creatine monohydrate in muscular dystrophies: A double-blind, placebo-controlled clinical study

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7 January 2021

Leukemia inhibitory factor ameliorates muscle fiber degeneration in the mdx mouse

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7 January 2021

Delay of muscle degeneration and necrosis in mdx mice by calpain inhibition.

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7 January 2021

Pre-clinical screening of drugs using the mdx mouse

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7 January 2021

Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children.

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7 January 2021

Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene

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7 January 2021

The dystrophin complex forms a mechanically strong link between the sarcolemma and costameric actin

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https://api.crossref.org/works/10.1016%2FS0165-6147%2800%2901770-3

7 January 2021

The artificial zinc finger coding gene 'Jazz' binds the utrophin promoter and activates transcription

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https://api.crossref.org/works/10.1016%2FS0165-6147%2800%2901770-3

7 January 2021

Expression of messenger RNA of the cardiac isoforms of troponin T and I in myopathic skeletal muscle

1 reference

https://api.crossref.org/works/10.1016%2FS0165-6147%2800%2901770-3

7 January 2021

10.1016/S0165-6147(00)01770-3

Identifiers

DOI

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1 August 2017

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