Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy. - Wikidata - wikimedia - TriplyDB

Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy.

Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy.

http://www.wikidata.org/entity/Q30503157

about

Spinal muscular atrophy: development and implementation of potential treatments Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)Skeletal muscle DNA damage precedes spinal motor neuron DNA damage in a mouse model of Spinal Muscular Atrophy (SMA)A large animal model of spinal muscular atrophy and correction of phenotype.Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy Conditionals by inversion provide a universal method for the generation of conditional alleles Collaboration for rare disease drug discovery research Temporal requirement for SMN in motoneuron development.Sodium vanadate combined with L-ascorbic acid delays disease progression, enhances motor performance, and ameliorates muscle atrophy and weakness in mice with spinal muscular atrophy.The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models.Pathological impact of SMN2 mis-splicing in adult SMA mice Requirement of enhanced Survival Motoneuron protein imposed during neuromuscular junction maturation.PTEN depletion decreases disease severity and modestly prolongs survival in a mouse model of spinal muscular atrophy.Emerging therapies and challenges in spinal muscular atrophy Spinal Muscular Atrophy: From Defective Chaperoning of snRNP Assembly to Neuromuscular Dysfunction.Defining the therapeutic window in a severe animal model of spinal muscular atrophy New therapeutic approaches to spinal muscular atrophy.A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse Antisense oligonucleotides for the treatment of spinal muscular atrophy.GEMINs: potential therapeutic targets for spinal muscular atrophy?Limited phenotypic effects of selectively augmenting the SMN protein in the neurons of a mouse model of severe spinal muscular atrophy.Spinal Muscular Atrophy Therapeutics: Where do we Stand?Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds Spinal muscular atrophy: diagnosis and management in a new therapeutic era Improvement of neuromuscular synaptic phenotypes without enhanced survival and motor function in severe spinal muscular atrophy mice selectively rescued in motor neurons Muscle-derived extracellular signal-regulated kinases 1 and 2 are required for the maintenance of adult myofibers and their neuromuscular junctions.Advances in therapeutic development for spinal muscular atrophy.Spinal muscular atrophy: journeying from bench to bedside Severe neuromuscular denervation of clinically relevant muscles in a mouse model of spinal muscular atrophy.Disease mechanisms and therapeutic approaches in spinal muscular atrophy.Understanding the experiences and needs of individuals with Spinal Muscular Atrophy and their parents: a qualitative study Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery Meeting Report: New Directions in the Biology and Disease of Skeletal Muscle 2014 SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA.Mutant superoxide dismutase 1 (SOD1), a cause of amyotrophic lateral sclerosis, disrupts the recruitment of SMN, the spinal muscular atrophy protein to nuclear Cajal bodies.Low levels of Survival Motor Neuron protein are sufficient for normal muscle function in the SMNΔ7 mouse model of SMA.TSUNAMI: an antisense method to phenocopy splicing-associated diseases in animals.Systemic, postsymptomatic antisense oligonucleotide rescues motor unit maturation delay in a new mouse model for type II/III spinal muscular atrophy.Survival motor neuron protein in motor neurons determines synaptic integrity in spinal muscular atrophy Behavioral and electrophysiological outcomes of tissue-specific Smn knockdown in Drosophila melanogaster.

P2860

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P2860

Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy.

http://www.wikidata.org/entity/Q30503157

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2011 nî lūn-bûn

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2011 թուականի Յուլիսին հրատարակուած գիտական յօդուած

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2011 թվականի հուլիսին հրատարակված գիտական հոդված

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2011年の論文

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2011年論文

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2011年論文

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2011年論文

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2011年論文

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2011年論文

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Postsymptomatic restoration of ...... evere spinal muscular atrophy.

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Postsymptomatic restoration of ...... evere spinal muscular atrophy.

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Postsymptomatic restoration of ...... evere spinal muscular atrophy.

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Postsymptomatic restoration of ...... evere spinal muscular atrophy.

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Journal of Clinical Investigation

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Postsymptomatic restoration of ...... severe spinal muscular atrophy

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P2093

Andrew J Murphy

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Cathleen M Lutz

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Darrick K Li

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David M Valenzuela

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Don Liu

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José Rojas

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Margaret L Winberg

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Melissa A Osborne

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Shingo Kariya

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Sunita Patruni

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P2860

A bacterial artificial chromosome library for sequencing the complete human genome

A novel nuclear structure containing the survival of motor neurons protein

Indoprofen upregulates the survival motor neuron protein through a cyclooxygenase-independent mechanism

High-throughput engineering of the mouse genome coupled with high-resolution expression analysis

Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients

Unrip is a component of SMN complexes active in snRNP assembly

Hydroxyurea enhances SMN2 gene expression in spinal muscular atrophy cells

SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy

Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery

Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy.

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3029-3041

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10.1172/JCI57291

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P433

8

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121

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Christopher E. Henderson

Livio Pellizzoni

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2011-07-25T00:00:00Z

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21785219

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spinal muscular atrophy

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3148744

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