Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models.
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Spinal Muscular AtrophyRepeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic responseTherapeutic Potential of Tricyclo-DNA antisense oligonucleotidesReport on the 3rd Ottawa International Conference on Neuromuscular Biology, Disease and Therapy - September 24-26, 2015, Ottawa, Canada.Protective effects of butyrate-based compounds on a mouse model for spinal muscular atrophy.Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophyEfficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide TreatmentIdentification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular AtrophySpinal Muscular Atrophy Therapeutics: Where do we Stand?Targeting SR proteins improves SMN expression in spinal muscular atrophy cells.Splicing regulation in spinal muscular atrophy by an RNA structure formed by long-distance interactions.Mechanistic principles of antisense targets for the treatment of spinal muscular atrophySplice-switching antisense oligonucleotides as therapeutic drugsML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in micePlastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophy.Spinal muscular atrophy--recent therapeutic advances for an old challenge.Developing therapies for spinal muscular atrophy.Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease.Pharmacology of Modulators of Alternative Splicing.Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.Advances in modeling and treating spinal muscular atrophyTranslational development of splice-modifying antisense oligomers.How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophyAntisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases.Modulation of nuclear REST by alternative splicing: a potential therapeutic target for Huntington's disease.Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis.Oligonucleotide therapies for disorders of the nervous system.SMN deficiency negatively impacts red pulp macrophages and spleen development in mouse models of spinal muscular atrophy.Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript.Blocking p62-dependent SMN degradation ameliorates spinal muscular atrophy disease phenotypes
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P2860
Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models.
description
2014 nî lūn-bûn
@nan
2014年の論文
@ja
2014年論文
@yue
2014年論文
@zh-hant
2014年論文
@zh-hk
2014年論文
@zh-mo
2014年論文
@zh-tw
2014年论文
@wuu
2014年论文
@zh
2014年论文
@zh-cn
name
Morpholino antisense oligonucl ...... phenotype in SMA mouse models.
@en
Morpholino antisense oligonucl ...... phenotype in SMA mouse models.
@nl
type
label
Morpholino antisense oligonucl ...... phenotype in SMA mouse models.
@en
Morpholino antisense oligonucl ...... phenotype in SMA mouse models.
@nl
prefLabel
Morpholino antisense oligonucl ...... phenotype in SMA mouse models.
@en
Morpholino antisense oligonucl ...... phenotype in SMA mouse models.
@nl
P2093
P2860
P356
P1476
Morpholino antisense oligonucl ...... phenotype in SMA mouse models.
@en
P2093
Abby M Lombardi
Amanda J Brehm
Arleigh K Atkinson
Christian L Lorson
Erkan Y Osman
Kate L Robbins
Madeline R Miller
P2860
P304
P356
10.1093/HMG/DDU198
P577
2014-04-29T00:00:00Z