Enzyme replacement therapy rescues weakness and improves muscle pathology in mice with X-linked myotubular myopathy.
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Intracellular Delivery of Proteins with Cell-Penetrating Peptides for Therapeutic Uses in Human DiseaseSkeletal Muscle Pathology in X-Linked Myotubular Myopathy: Review With Cross-Species ComparisonsGait characteristics in a canine model of X-linked myotubular myopathyPathophysiological concepts in the congenital myopathies: blurring the boundaries, sharpening the focusUltrasound assessment of the diaphragm: Preliminary study of a canine model of X-linked myotubular myopathy.Differential muscle hypertrophy is associated with satellite cell numbers and Akt pathway activation following activin type IIB receptor inhibition in Mtm1 p.R69C miceGene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.Tissue triage and freezing for models of skeletal muscle disease.Triadopathies: an emerging class of skeletal muscle diseases.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.Treatment with ActRIIB-mFc Produces Myofiber Growth and Improves Lifespan in the Acta1 H40Y Murine Model of Nemaline Myopathy.Dynamin 2 the rescue for centronuclear myopathy.A nucleolytic lupus autoantibody is toxic to BRCA2-deficient cancer cells.Getting across the cell membrane: an overview for small molecules, peptides, and proteins.A natural history study of X-linked myotubular myopathy.Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease.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.Expression of the neuropathy-associated MTMR2 gene rescues MTM1-associated myopathy.Affected female carriers of MTM1 mutations display a wide spectrum of clinical and pathological involvement: delineating diagnostic clues.ECM-Related Myopathies and Muscular Dystrophies: Pros and Cons of Protein Therapies.Myostatin inhibition using mRK35 produces skeletal muscle growth and tubular aggregate formation in wild type and TgACTA1D286G nemaline myopathy mice.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.Treating pediatric neuromuscular disorders: The future is now.Review of X-linked syndromes with arthrogryposis or early contractures-aid to diagnosis and pathway identification.Centronuclear myopathies under attack: A plethora of therapeutic targets
P2860
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P2860
Enzyme replacement therapy rescues weakness and improves muscle pathology in mice with X-linked myotubular myopathy.
description
2013 nî lūn-bûn
@nan
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
2013年论文
@zh
2013年论文
@zh-cn
name
Enzyme replacement therapy res ...... X-linked myotubular myopathy.
@ast
Enzyme replacement therapy res ...... X-linked myotubular myopathy.
@en
type
label
Enzyme replacement therapy res ...... X-linked myotubular myopathy.
@ast
Enzyme replacement therapy res ...... X-linked myotubular myopathy.
@en
prefLabel
Enzyme replacement therapy res ...... X-linked myotubular myopathy.
@ast
Enzyme replacement therapy res ...... X-linked myotubular myopathy.
@en
P2093
P2860
P356
P1476
Enzyme replacement therapy res ...... h X-linked myotubular myopathy
@en
P2093
Anna Buj-Bello
Christopher R Pierson
Cynthia P Hsu
Dustin Armstrong
Jeffrey J Widrick
Marissa G Viola
Michael O'Callaghan
Michael W Lawlor
Robert W Grange
P2860
P304
P356
10.1093/HMG/DDT003
P577
2013-01-09T00:00:00Z