Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.

Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.

Item
http://www.wikidata.org/entity/Q34902920
Molecular Mechanisms of Neurodegeneration in Spinal Muscular AtrophySpinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.Molecular Basis and Therapeutic Strategies to Rescue Factor IX Variants That Affect Splicing and Protein FunctionTherapeutic activity of modified U1 core spliceosomal particlesViral Vector-Mediated Antisense Therapy for Genetic Diseases.Current and future therapies for inherited cholestatic liver diseases.Exon-Specific U1s Correct SPINK5 Exon 11 Skipping Caused by a Synonymous Substitution that Affects a Bifunctional Splicing Regulatory Element.Faulty RNA splicing: consequences and therapeutic opportunities in brain and muscle disorders.Correction of a Cystic Fibrosis Splicing Mutation by Antisense Oligonucleotides.An Exon-Specific U1snRNA Induces a Robust Factor IX Activity in Mice Expressing Multiple Human FIX Splicing Mutants.Activation of a cryptic 5' splice site reverses the impact of pathogenic splice site mutations in the spinal muscular atrophy gene.Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis.Exon Specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a Familial Dysautonomia mouse model.Intronic PAH gene mutations cause a splicing defect by a novel mechanism involving U1snRNP binding downstream of the 5' splice site.A new biomarker candidate for spinal muscular atrophy: Identification of a peripheral blood cell population capable of monitoring the level of survival motor neuron protein
P2860

Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.

Item
http://www.wikidata.org/entity/Q34902920
description
2014 nî lūn-bûn
@nan
2014 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2014 թվականի դեկտեմբերին հրատարակված գիտական հոդված
@hy
2014年の論文
@ja
2014年学术文章
@wuu
2014年学术文章
@zh-cn
2014年学术文章
@zh-hans
2014年学术文章
@zh-my
2014年学术文章
@zh-sg
2014年學術文章
@yue
name
Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.
@ast
Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.
@en
Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.
@nl
type
label
Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.
@ast
Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.
@en
Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.
@nl
prefLabel
Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.
@ast
Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.
@en
Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.
@nl
P1433
P1476
P2093
P2860
P304
P31
P356
P407
P433
P478
P50
P577
P5875
P698
P932
P356
P1433
P1476
Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.
@en
P2093
P2860
P304
P31
P356
10.1016/J.AJHG.2014.12.009
P407
P433
P478
P50
P577
P5875
P698
P932