Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy
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Title
Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy
Authors
Keywords
Mouse models, Fibroblasts, Oligonucleotides, Transfection, Reverse transcriptase-polymerase chain reaction, Introns, Necrosis, Protein expression
Journal
PLoS One
Volume 8, Issue 4, Pages e62114
Publisher
Public Library of Science (PLoS)
Online
2013-04-23
DOI
10.1371/journal.pone.0062114
References
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Note: Only part of the references are listed.- A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse
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- Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy
- (2010) C. F. Valori et al. Science Translational Medicine
- Effect of diet on the survival and phenotype of a mouse model for spinal muscular atrophy
- (2009) Matthew E.R. Butchbach et al. BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS
- Vascular Perfusion Abnormalities in Infants with Spinal Muscular Atrophy
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- Rational Design of Antisense Oligomers to Induce Dystrophin Exon Skipping
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- Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons
- (2009) Sandra Duque et al. MOLECULAR THERAPY
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- Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice
- (2008) Yimin Hua et al. AMERICAN JOURNAL OF HUMAN GENETICS
- Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect
- (2008) T. O. Gavrilina et al. HUMAN MOLECULAR GENETICS
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- (2008) B. G. Burnett et al. MOLECULAR AND CELLULAR BIOLOGY
- Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
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