Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice. - Wikidata - wikimedia - TriplyDB

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.

http://www.wikidata.org/entity/Q36718923

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Development of a single vector system that enhances trans-splicing of SMN2 transcripts Modeling spinal muscular atrophy in Drosophila Drug treatment for spinal muscular atrophy types II and III Drug treatment for spinal muscular atrophy types II and III The splicing regulator Sam68 binds to a novel exonic splicing silencer and functions in SMN2 alternative splicing in spinal muscular atrophy Cholinergic-associated loss of hnRNP-A/B in Alzheimer's disease impairs cortical splicing and cognitive function in mice Differential 3' splice site recognition of SMN1 and SMN2 transcripts by U2AF and U2 snRNP Spinal muscular atrophy: from gene discovery to clinical trials Synthetic biology and therapeutic strategies for the degenerating brain: Synthetic biology approaches can transform classical cell and gene therapies, to provide new cures for neurodegenerative diseases Oligonucleotide Therapies: The Past and the Present Antisense oligonucleotides: treating neurodegeneration at the level of RNA Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)Splicing therapy for neuromuscular disease A splice site mutation in laminin-α2 results in a severe muscular dystrophy and growth abnormalities in zebrafish Progress toward therapy with antisense-mediated splicing modulation Spinal Muscular Atrophy Applications of Cas9 as an RNA-programmed RNA-binding protein A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation Evaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study Synthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicing SMN in spinal muscular atrophy and snRNP biogenesis CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy.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.Pathological impact of SMN2 mis-splicing in adult SMA mice Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing A method of predicting changes in human gene splicing induced by genetic variants in context of cis-acting elements Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.The alternative splicing repressors hnRNP A1/A2 and PTB influence pyruvate kinase isoform expression and cell metabolism.The notorious R.N.A. in the spotlight - drug or target for the treatment of disease Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts Spinal muscular atrophy: new and emerging insights from model mice An antisense microwalk reveals critical role of an intronic position linked to a unique long-distance interaction in pre-mRNA splicing Spinal muscular atrophy: mechanisms and therapeutic strategies.Intron 7 conserved sequence elements regulate the splicing of the SMN genes.Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model Splicing of the Survival Motor Neuron genes and implications for treatment of SMA

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P2860

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.

http://www.wikidata.org/entity/Q36718923

description

2008 nî lūn-bûn

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2008年の論文

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2008年論文

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2008年論文

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2008年論文

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name

Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.

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Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.

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type

label

Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.

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Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.

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prefLabel

Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.

@ast

Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.

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J.AJHG.2008.01.014

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American Journal of Human Genetics

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Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.

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P2093

C Frank Bennett

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Hazeem L Okunola

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Timothy A Vickers

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Yimin Hua

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P2860

HnRNP G and Tra2beta: opposite effects on splicing matched by antagonism in RNA binding

hnRNP-G promotes exon 7 inclusion of survival motor neuron (SMN) via direct interaction with Htra2-beta1

Reversal of the cellular phenotype in the premature aging disease Hutchinson-Gilford progeria syndrome

Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides

A combinatorial code for splicing silencing: UAGG and GGGG motifs

Crystal structure of the two-RRM domain of hnRNP A1 (UP1) complexed with single-stranded telomeric DNA

SRp30c-dependent stimulation of survival motor neuron (SMN) exon 7 inclusion is facilitated by a direct interaction with hTra2 beta 1

An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA

TDP-43 binds heterogeneous nuclear ribonucleoprotein A/B through its C-terminal tail: an important region for the inhibition of cystic fibrosis transmembrane conductance regulator exon 9 splicing

Association of missense and 5'-splice-site mutations in tau with the inherited dementia FTDP-17

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834-848

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