Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy
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Is spinal muscular atrophy a disease of the motor neurons only: pathogenesis and therapeutic implications?Spinal muscular atrophy: development and implementation of potential treatmentsMolecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)Sleep and sleep disorders in rare hereditary diseases: a reminder for the pediatrician, pediatric and adult neurologist, general practitioner, and sleep specialistTargeting mRNA for the treatment of facioscapulohumeral muscular dystrophyProtective effects of butyrate-based compounds on a mouse model for 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.Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular AtrophyAssays for the identification and prioritization of drug candidates for spinal muscular atrophyAntisense oligonucleotides for the treatment of spinal muscular atrophy.Spinal Muscular Atrophy Therapeutics: Where do we Stand?Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity.ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.Antisense therapy in neurologyState-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications.Spinal muscular atrophy: from tissue specificity to therapeutic strategiesAdvances 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.Splice-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.hnRNP A1: the Swiss army knife of gene expression.Human RNAi pathway: crosstalk with organelles and cells.Spinal muscular atrophy--recent therapeutic advances for an old challenge.Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease.Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.Modeling the early phenotype at the neuromuscular junction of spinal muscular atrophy using patient-derived iPSCs.How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophyMaking sense of antisense oligonucleotides: A narrative review.Intracerebroventricular Delivery in Mice for Motor Neuron Diseases.Analysis of Azithromycin Monohydrate as a Single or a Combinatorial Therapy in a Mouse Model of Severe Spinal Muscular Atrophy.Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript.
P2860
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P2860
Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy
description
2013 nî lūn-bûn
@nan
2013 թուականի Ապրիլին հրատարակուած գիտական յօդուած
@hyw
2013 թվականի ապրիլին հրատարակված գիտական հոդված
@hy
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
name
Improved antisense oligonucleo ...... nt for spinal muscular atrophy
@ast
Improved antisense oligonucleo ...... nt for spinal muscular atrophy
@en
Improved antisense oligonucleo ...... nt for spinal muscular atrophy
@nl
type
label
Improved antisense oligonucleo ...... nt for spinal muscular atrophy
@ast
Improved antisense oligonucleo ...... nt for spinal muscular atrophy
@en
Improved antisense oligonucleo ...... nt for spinal muscular atrophy
@nl
prefLabel
Improved antisense oligonucleo ...... nt for spinal muscular atrophy
@ast
Improved antisense oligonucleo ...... nt for spinal muscular atrophy
@en
Improved antisense oligonucleo ...... nt for spinal muscular atrophy
@nl
P2093
P2860
P1433
P1476
Improved antisense oligonucleo ...... nt for spinal muscular atrophy
@en
P2093
Arthur H M Burghes
Chalermchai Mitrpant
Francesco Muntoni
Haiyan Zhou
Loren Price
Paul Porensky
Steve D Wilton
Sue Fletcher
P2860
P304
P356
10.1371/JOURNAL.PONE.0062114
P407
P577
2013-04-22T00:00:00Z