A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice
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Spinal muscular atrophy: development and implementation of potential treatmentsA chemical view of oligonucleotides for exon skipping and related drug applicationsAntisense oligonucleotides: treating neurodegeneration at the level of RNAMolecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)Targeting mRNA for the treatment of facioscapulohumeral muscular dystrophyRepeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic responseSelective Neuromuscular Denervation in Taiwanese Severe SMA Mouse Can Be Reversed by Morpholino Antisense OligonucleotidesProtective effects of butyrate-based compounds on a mouse model for spinal muscular atrophy.Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide TreatmentPharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular AtrophyLNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblastsA short antisense oligonucleotide ameliorates symptoms of severe mouse models of spinal muscular atrophyAntisense oligonucleotides for the treatment of spinal muscular atrophy.GEMINs: potential therapeutic targets for spinal muscular atrophy?Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity.Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophySpinal muscular atrophy: from tissue specificity to therapeutic strategiesMotor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse modelsAdvances in therapeutic development for spinal muscular atrophy.Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment.Mechanistic principles of antisense targets for the treatment of spinal muscular atrophyMorpholino-mediated SOD1 reduction ameliorates an amyotrophic lateral sclerosis disease phenotype.An intronic structure enabled by a long-distance interaction serves as a novel target for splicing correction in spinal muscular atrophySplice-switching antisense oligonucleotides as therapeutic drugsAltered Levels of MicroRNA-9, -206, and -132 in Spinal Muscular Atrophy and Their Response to Antisense Oligonucleotide TherapySpinal muscular atrophy: an update on therapeutic progressSurvival Motor Neuron (SMN) protein is required for normal mouse liver development.Antisense oligonucleotide mediated therapy of spinal muscular atrophyAlternatively spliced mu opioid receptor C termini impact the diverse actions of morphine.Small RNA drugs for prion disease: a new frontier.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.Sequence-defined polymers for the delivery of oligonucleotides.Investigational therapies for the treatment of spinal muscular atrophy.Spinal muscular atrophy--recent therapeutic advances for an old challenge.Novel splice-switching oligonucleotide promotes BRCA1 aberrant splicing and susceptibility to PARP inhibitor action.Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease.Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.
P2860
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P2860
A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice
description
2013 nî lūn-bûn
@nan
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
2013年论文
@zh
2013年论文
@zh-cn
name
A novel morpholino oligomer ta ...... scular atrophy transgenic mice
@en
A novel morpholino oligomer ta ...... cular atrophy transgenic mice.
@nl
type
label
A novel morpholino oligomer ta ...... scular atrophy transgenic mice
@en
A novel morpholino oligomer ta ...... cular atrophy transgenic mice.
@nl
prefLabel
A novel morpholino oligomer ta ...... scular atrophy transgenic mice
@en
A novel morpholino oligomer ta ...... cular atrophy transgenic mice.
@nl
P2093
P2860
P356
P1433
P1476
A novel morpholino oligomer ta ...... scular atrophy transgenic mice
@en
P2093
Francesco Muntoni
Haiyan Zhou
Ian C Eperon
Jennifer Morgan
Loren Price
Narinder Janghra
Rachel L Dickinson
Stephen D Wilton
P2860
P304
P356
10.1089/HUM.2012.211
P577
2013-03-06T00:00:00Z