Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy.
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The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophy5p deletions: Current knowledge and future directions.Spinal muscular atrophy: development and implementation of potential treatmentsApplicability of histone deacetylase inhibition for the treatment of spinal muscular atrophyMolecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy.A large animal model of spinal muscular atrophy and correction of phenotype.Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model.Spinal Muscular AtrophyProlactin increases SMN expression and survival in a mouse model of severe spinal muscular atrophy via the STAT5 pathway.Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic responseEvaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical studyCandidate proteins, metabolites and transcripts in the Biomarkers for Spinal Muscular Atrophy (BforSMA) clinical studyPostsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy.Requirement of enhanced Survival Motoneuron protein imposed during neuromuscular junction maturation.PTEN depletion decreases disease severity and modestly prolongs survival in a mouse model of spinal muscular atrophy.Protective effects of butyrate-based compounds on a mouse model for spinal muscular atrophy.Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 SplicingDefining the therapeutic window in a severe animal model of spinal muscular atrophyAssays for the identification and prioritization of drug candidates for spinal muscular atrophyTranscriptome profiling of spinal muscular atrophy motor neurons derived from mouse embryonic stem cellsReduced survival of motor neuron (SMN) protein in motor neuronal progenitors functions cell autonomously to cause spinal muscular atrophy in model mice expressing the human centromeric (SMN2) geneAntisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.Drug discovery and development for spinal muscular atrophy: lessons from screening approaches and future challenges for clinical development.Temporal requirement for high SMN expression in SMA miceSystemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disordersNew therapeutic approaches to spinal muscular atrophy.A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouseTherapeutic strategies for the treatment of spinal muscular atrophy.SMN-inducing compounds for the treatment of spinal muscular atrophy.Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice.Antisense oligonucleotides for the treatment of spinal muscular atrophy.Early functional impairment of sensory-motor connectivity in a mouse model of spinal muscular atrophy.A single intravenous AAV9 injection mediates bilateral gene transfer to the adult mouse retina.Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophyEnhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compoundsSpinal muscular atrophy: diagnosis and management in a new therapeutic eraA cell system for phenotypic screening of modifiers of SMN2 gene expression and function.Spinal muscular atrophy: from tissue specificity to therapeutic strategiesImproving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
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P2860
Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy.
description
2010 nî lūn-bûn
@nan
2010年の論文
@ja
2010年学术文章
@wuu
2010年学术文章
@zh
2010年学术文章
@zh-cn
2010年学术文章
@zh-hans
2010年学术文章
@zh-my
2010年学术文章
@zh-sg
2010年學術文章
@yue
2010年學術文章
@zh-hant
name
Systemic delivery of scAAV9 ex ...... el of spinal muscular atrophy.
@en
Systemic delivery of scAAV9 ex ...... el of spinal muscular atrophy.
@nl
type
label
Systemic delivery of scAAV9 ex ...... el of spinal muscular atrophy.
@en
Systemic delivery of scAAV9 ex ...... el of spinal muscular atrophy.
@nl
prefLabel
Systemic delivery of scAAV9 ex ...... el of spinal muscular atrophy.
@en
Systemic delivery of scAAV9 ex ...... el of spinal muscular atrophy.
@nl
P2860
P50
P1476
Systemic delivery of scAAV9 ex ...... el of spinal muscular atrophy.
@en
P2093
Chiara F Valori
Matthew Wyles
P2860
P304
P356
10.1126/SCITRANSLMED.3000830
P407
P577
2010-06-01T00:00:00Z