Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.
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Mapping in vivo protein-RNA interactions at single-nucleotide resolution from HITS-CLIP datamRNA trans-splicing in gene therapy for genetic diseasesSpinal muscular atrophy: from gene discovery to clinical trialsSpinal muscular atrophy: development and implementation of potential treatmentsTrans-splicingSpinal Muscular AtrophyRepair of Mybpc3 mRNA by 5'-trans-splicing in a Mouse Model of Hypertrophic CardiomyopathyMYBPC3 in hypertrophic cardiomyopathy: from mutation identification to RNA-based correctionSMN in spinal muscular atrophy and snRNP biogenesisPostsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy.Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.Spinal muscular atrophy: mechanisms and therapeutic strategies.Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse modelAntisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.Delivery of therapeutic agents through intracerebroventricular (ICV) and intravenous (IV) injection in mice.SMN-inducing compounds for the treatment of spinal muscular atrophy.Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compoundsRepair of rhodopsin mRNA by spliceosome-mediated RNA trans-splicing: a new approach for autosomal dominant retinitis pigmentosa.Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA.Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN deliveryTargeting RNA splicing for disease therapy.Reprogramming the Dynamin 2 mRNA by Spliceosome-mediated RNA Trans-splicing.Spinal muscular atrophy: a timely review.Targeting RNA-splicing for SMA treatment.Genetic therapy for the nervous system.RNA splicing: disease and therapy.RNA-based therapeutic approaches for coagulation factor deficiencies.Mouse models of SMA: tools for disease characterization and therapeutic development.Exon identity crisis: disease-causing mutations that disrupt the splicing code.Trans-splicing improvement by the combined application of antisense strategies.Construction and validation of an RNA trans-splicing molecule suitable to repair a large number of COL7A1 mutations.Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.Dysferlin rescue by spliceosome-mediated pre-mRNA trans-splicing targeting introns harbouring weakly defined 3' splice sites.Cis-splicing and translation of the pre-trans-splicing molecule combine with efficiency in spliceosome-mediated RNA trans-splicingCorrection of the Middle Eastern M712T mutation causing GNE myopathy by trans-splicing.Targeting Splicing in the Treatment of Human DiseaseThe design and optimization of RNA trans-splicing molecules for skin cancer therapy.Optimization of trans-Splicing for Huntington's Disease RNA Therapy.Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice.An RNA-targeted therapy for dystrophic epidermolysis bullosa.
P2860
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P2860
Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.
description
2010 nî lūn-bûn
@nan
2010 թուականի Յունուարին հրատարակուած գիտական յօդուած
@hyw
2010 թվականի հունվարին հրատարակված գիտական հոդված
@hy
2010年の論文
@ja
2010年論文
@yue
2010年論文
@zh-hant
2010年論文
@zh-hk
2010年論文
@zh-mo
2010年論文
@zh-tw
2010年论文
@wuu
name
Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.
@ast
Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.
@en
type
label
Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.
@ast
Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.
@en
prefLabel
Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.
@ast
Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.
@en
P2860
P1476
Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.
@en
P2093
Christian L Lorson
Tristan H Coady
P2860
P304
P356
10.1523/JNEUROSCI.4489-09.2010
P407
P577
2010-01-01T00:00:00Z