Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy. - Wikidata - thesis-toulmin - TriplyDB

Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.

Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.

http://www.wikidata.org/entity/Q33926944

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Skeletal Muscle Pathology in X-Linked Myotubular Myopathy: Review With Cross-Species Comparisons Respiratory assessment in centronuclear myopathies Validity of a Neurological Scoring System for Canine X-Linked Myotubular Myopathy Gait characteristics in a canine model of X-linked myotubular myopathy Pathophysiological concepts in the congenital myopathies: blurring the boundaries, sharpening the focus Ultrasound assessment of the diaphragm: Preliminary study of a canine model of X-linked myotubular myopathy.PIK3C2B inhibition improves function and prolongs survival in myotubular myopathy animal models Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine.Guiding intramuscular diaphragm injections using real-time ultrasound and electromyography.Clinical phenotype of X-linked myotubular myopathy in Labrador Retriever puppies.X-linked myotubular myopathy in Rottweiler dogs is caused by a missense mutation in Exon 11 of the MTM1 gene Triadopathies: an emerging class of skeletal muscle diseases.Duchenne muscular dystrophy gene therapy in the canine model.Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy.Muscle pathology, limb strength, walking gait, respiratory function and neurological impairment establish disease progression in the p.N155K canine model of X-linked myotubular myopathy.The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer.Gene therapy in monogenic congenital myopathies.A natural history study of X-linked myotubular myopathy.Systemic delivery of adeno-associated viral vectors.Gene therapy in myotubular myopathy: promising progress and future directions.Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry.Long-term effects of systemic gene therapy in a canine model of myotubular myopathy.Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.[The Emeritus Dinosaur's viewpoint - The road to hell of gene therapists is paved with POC].Exome sequencing reveals a nebulin nonsense mutation in a dog model of nemaline myopathy.Suspected congenital centronuclear myopathy in an Arabian-cross foal.Noninvasive Assessment of Neuromuscular Disease in Dogs: Use of the 6-minute Walk Test to Assess Submaximal Exercise Tolerance in Dogs with Centronuclear Myopathy.Gene replacement rescues severe muscle pathology and prolongs survival in myotubularin-deficient mice and dogs.In Vitro Assays to Determine Skeletal Muscle Physiologic Function.Systemic AAV Gene Therapy Close to Clinical Trials for Several Neuromuscular Diseases.Long-term effects of systemic gene therapy On gait in a canine model of myotubular myopathy.Affected female carriers of MTM1 mutations display a wide spectrum of clinical and pathological involvement: delineating diagnostic clues.AAV6 Vector Production and Purification for Muscle Gene Therapy.Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches.A multicenter, retrospective medical record review of X-linked myotubular myopathy: The recensus study.Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice.Single Intramuscular Injection of AAV-shRNA Reduces DNM2 and Prevents Myotubular Myopathy in Mice.Treating pediatric neuromuscular disorders: The future is now.Centronuclear myopathies under attack: A plethora of therapeutic targets Tamoxifen prolongs survival and alleviates symptoms in mice with fatal X-linked myotubular myopathy

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Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.

http://www.wikidata.org/entity/Q33926944

description

2014 nî lūn-bûn

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2014 թուականի Յունուարին հրատարակուած գիտական յօդուած

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2014 թվականի հունվարին հրատարակված գիտական հոդված

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2014年の論文

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2014年学术文章

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2014年學術文章

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Gene therapy prolongs survival ...... models of myotubular myopathy.

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Gene therapy prolongs survival ...... models of myotubular myopathy.

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Gene therapy prolongs survival ...... models of myotubular myopathy.

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Gene therapy prolongs survival ...... models of myotubular myopathy.

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Gene therapy prolongs survival ...... models of myotubular myopathy.

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Gene therapy prolongs survival ...... models of myotubular myopathy.

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Science Translational Medicine

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Gene therapy prolongs survival ...... models of myotubular myopathy

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Alban Vignaud

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Anna Buj-Bello

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Branden E Rider

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Christelle Moal

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Erin Mitchell

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J Koudy Williams

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P2860

Characterization of the myotubularin dual specificity phosphatase gene family from yeast to human

Safety and efficacy of gene transfer for Leber's congenital amaurosis

Loss of myotubularin function results in T-tubule disorganization in zebrafish and human myotubular myopathy

Effect of gene therapy on visual function in Leber's congenital amaurosis

A gene mutated in X-linked myotubular myopathy defines a new putative tyrosine phosphatase family conserved in yeast

AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis

The lipid phosphatase myotubularin is essential for skeletal muscle maintenance but not for myogenesis in mice

Site-specific Mtm1 mutagenesis by an AAV-Cre vector reveals that myotubularin is essential in adult muscle

AAV2 gene therapy readministration in three adults with congenital blindness.

A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells

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