Dual masking of specific negative splicing regulatory elements resulted in maximal exon 7 inclusion of SMN2 gene. - Wikidata - wikimedia - TriplyDB

Dual masking of specific negative splicing regulatory elements resulted in maximal exon 7 inclusion of SMN2 gene.

Dual masking of specific negative splicing regulatory elements resulted in maximal exon 7 inclusion of SMN2 gene.

http://www.wikidata.org/entity/Q37696051

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Advanced In vivo Use of CRISPR/Cas9 and Anti-sense DNA Inhibition for Gene Manipulation in the Brain ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy MYC regulates the core pre-mRNA splicing machinery as an essential step in lymphomagenesis.Diverse role of survival motor neuron protein.How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases.Peptide nanoparticle delivery of charge-neutral splice-switching morpholino oligonucleotides.Application of urine cells in drug intervention for spinal muscular atrophy.Induced-Decay of Glycine Decarboxylase Transcripts as an Anticancer Therapeutic Strategy for Non-Small-Cell Lung Carcinoma.Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis.

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Dual masking of specific negative splicing regulatory elements resulted in maximal exon 7 inclusion of SMN2 gene.

http://www.wikidata.org/entity/Q37696051

description

article científic

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article scientifique

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articolo scientifico

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artigo científico

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bilimsel makale

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scientific article published on 09 December 2013

@en

vedecký článok

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vetenskaplig artikel

@sv

videnskabelig artikel

@da

vědecký článek

@cs

name

Dual masking of specific negat ...... exon 7 inclusion of SMN2 gene.

@en

Dual masking of specific negat ...... exon 7 inclusion of SMN2 gene.

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type

label

Dual masking of specific negat ...... exon 7 inclusion of SMN2 gene.

@en

Dual masking of specific negat ...... exon 7 inclusion of SMN2 gene.

@nl

prefLabel

Dual masking of specific negat ...... exon 7 inclusion of SMN2 gene.

@en

Dual masking of specific negat ...... exon 7 inclusion of SMN2 gene.

@nl

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Molecular Therapy

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Dual masking of specific negat ...... exon 7 inclusion of SMN2 gene.

@en

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Keng Boon Wee

http://www.w3.org/2001/XMLSchema#string

Peng Wen Pao

http://www.w3.org/2001/XMLSchema#string

Woon Chee Yee

http://www.w3.org/2001/XMLSchema#string

Zacharias Aloysius Dwi Pramono

http://www.w3.org/2001/XMLSchema#string

P2860

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

Synonymous mutations in CFTR exon 12 affect splicing and are not neutral in evolution.

Crystal structure of an 82-nucleotide RNA-DNA complex formed by the 10-23 DNA enzyme

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

Evidence for a modifying pathway in SMA discordant families: reduced SMN level decreases the amount of its interacting partners and Htra2-beta1

A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy

Correction of SMN2 Pre-mRNA splicing by antisense U7 small nuclear RNAs

Identification and characterization of a spinal muscular atrophy-determining gene

Expansion of the eukaryotic proteome by alternative splicing

A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

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854-861

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scholarly article

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10.1038/MT.2013.276

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4

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22

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2013-12-09T00:00:00Z

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3982506

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