Spinal muscular atrophy: journeying from bench to bedside - Wikidata - awgldk - TriplyDB

Spinal muscular atrophy: journeying from bench to bedside

Spinal muscular atrophy: journeying from bench to bedside

http://www.wikidata.org/entity/Q35340133

about

Emerging therapies and challenges in spinal muscular atrophy ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.Spinal muscular atrophy: from tissue specificity to therapeutic strategies Splicing regulation in spinal muscular atrophy by an RNA structure formed by long-distance interactions.Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy α-COP binding to the survival motor neuron protein SMN is required for neuronal process outgrowth Severe impairment of male reproductive organ development in a low SMN expressing mouse model of spinal muscular atrophy ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice SMN - A chaperone for nuclear RNP social occasions?Diverse role of survival motor neuron protein.How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy TIA1 is a gender-specific disease modifier of a mild mouse model of spinal muscular atrophy.Neurogenetic disease: genes, mechanisms, and future promise.Gender-Specific Amelioration of SMA Phenotype upon Disruption of a Deep Intronic Structure by an Oligonucleotide.

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P2860

Spinal muscular atrophy: journeying from bench to bedside

http://www.wikidata.org/entity/Q35340133

description

2014 nî lūn-bûn

@nan

2014 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած

@hyw

2014 թվականի հոտեմբերին հրատարակված գիտական հոդված

@hy

2014年の論文

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2014年論文

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2014年論文

@zh-hant

2014年論文

@zh-hk

2014年論文

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2014年論文

@zh-tw

2014年论文

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name

Spinal muscular atrophy: journeying from bench to bedside

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Spinal muscular atrophy: journeying from bench to bedside

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Spinal muscular atrophy: journeying from bench to bedside

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Spinal muscular atrophy: journeying from bench to bedside

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Spinal muscular atrophy: journeying from bench to bedside

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Spinal muscular atrophy: journeying from bench to bedside

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S13311-014-0293-Y

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Neurotherapeutics

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Spinal muscular atrophy: journeying from bench to bedside

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Jeong-Ki Kim

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Tomoyuki Awano

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Umrao R Monani

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P2860

The spinal muscular atrophy disease gene product, SMN, and its associated protein SIP1 are in a complex with spliceosomal snRNP proteins

The SMN-SIP1 complex has an essential role in spliceosomal snRNP biogenesis

A novel nuclear structure containing the survival of motor neurons protein

Stem cell transplantation for motor neuron disease: current approaches and future perspectives

Synaptic defects in the spinal and neuromuscular circuitry in a mouse model of spinal muscular atrophy

Specific interaction of Smn, the spinal muscular atrophy determining gene product, with hnRNP-R and gry-rbp/hnRNP-Q: a role for Smn in RNA processing in motor axons?

Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens

LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate

The changing natural history of spinal muscular atrophy type 1

Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy

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s13311-014-0293-y

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786-795

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10.1007/S13311-014-0293-Y

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4

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11

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2014-10-01T00:00:00Z

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spinal muscular atrophy

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