A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse
出版年份 2011 全文链接
标题
A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse
作者
关键词
-
出版物
HUMAN MOLECULAR GENETICS
Volume 21, Issue 7, Pages 1625-1638
出版商
Oxford University Press (OUP)
发表日期
2011-12-21
DOI
10.1093/hmg/ddr600
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注意:仅列出部分参考文献,下载原文获取全部文献信息。- Temporal requirement for high SMN expression in SMA mice
- (2011) T. T. Le et al. HUMAN MOLECULAR GENETICS
- Repeat-Dose Toxicology Evaluation in Cynomolgus Monkeys of AVI-4658, a Phosphorodiamidate Morpholino Oligomer (PMO) Drug for the Treatment of Duchenne Muscular Dystrophy
- (2011) Peter Sazani et al. INTERNATIONAL JOURNAL OF TOXICOLOGY
- Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy
- (2011) Cathleen M. Lutz et al. JOURNAL OF CLINICAL INVESTIGATION
- Prolactin increases SMN expression and survival in a mouse model of severe spinal muscular atrophy via the STAT5 pathway
- (2011) Faraz Farooq et al. JOURNAL OF CLINICAL INVESTIGATION
- Multiplex digital PCR: breaking the one target per color barrier of quantitative PCR
- (2011) Qun Zhong et al. LAB ON A CHIP
- Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
- (2011) Sebahattin Cirak et al. LANCET
- Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model
- (2011) Yimin Hua et al. NATURE
- Targeted Skipping of Human Dystrophin Exons in Transgenic Mouse Model Systemically for Antisense Drug Development
- (2011) Bo Wu et al. PLoS One
- Newborn and carrier screening for spinal muscular atrophy
- (2010) Thomas W. Prior et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Zebrafish as a model for vertebrate hematopoiesis
- (2010) Felix Ellett et al. CURRENT OPINION IN PHARMACOLOGY
- Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
- (2010) Y. Hua et al. GENES & DEVELOPMENT
- Antisense oligonucleotides and spinal muscular atrophy: skipping along
- (2010) A. H. M. Burghes et al. GENES & DEVELOPMENT
- Arrhythmia and cardiac defects are a feature of spinal muscular atrophy model mice
- (2010) Christopher R. Heier et al. HUMAN MOLECULAR GENETICS
- Cardiac defects contribute to the pathology of spinal muscular atrophy models
- (2010) Monir Shababi et al. HUMAN MOLECULAR GENETICS
- Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery
- (2010) Adam K. Bevan et al. HUMAN MOLECULAR GENETICS
- CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy
- (2010) Marco A. Passini et al. JOURNAL OF CLINICAL INVESTIGATION
- One-year Treatment of Morpholino Antisense Oligomer Improves Skeletal and Cardiac Muscle Functions in Dystrophic mdx Mice
- (2010) Bo Wu et al. MOLECULAR THERAPY
- Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
- (2010) Kevin D Foust et al. NATURE BIOTECHNOLOGY
- A Positive Modifier of Spinal Muscular Atrophy in the SMN2 Gene
- (2009) Thomas W. Prior et al. AMERICAN JOURNAL OF HUMAN GENETICS
- Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs
- (2009) Toshifumi Yokota et al. ANNALS OF NEUROLOGY
- Effects of 2,4-diaminoquinazoline derivatives on SMN expression and phenotype in a mouse model for spinal muscular atrophy
- (2009) Matthew E.R. Butchbach et al. HUMAN MOLECULAR GENETICS
- Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy
- (2009) T. D. Baughan et al. HUMAN MOLECULAR GENETICS
- A SMN missense mutation complements SMN2 restoring snRNPs and rescuing SMA mice
- (2009) Eileen Workman et al. HUMAN MOLECULAR GENETICS
- A rareSMN2variant in a previously unrecognized composite splicing regulatory element induces exon 7 inclusion and reduces the clinical severity of spinal muscular atrophy
- (2009) Myriam Vezain et al. HUMAN MUTATION
- Oligonucleotide-Mediated Survival of Motor Neuron Protein Expression in CNS Improves Phenotype in a Mouse Model of Spinal Muscular Atrophy
- (2009) J. H. Williams et al. JOURNAL OF NEUROSCIENCE
- Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
- (2009) Maria Kinali et al. LANCET NEUROLOGY
- Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?
- (2009) Arthur H. M. Burghes et al. NATURE REVIEWS NEUROSCIENCE
- Alternative Splicing Events Are a Late Feature of Pathology in a Mouse Model of Spinal Muscular Atrophy
- (2009) Dirk Bäumer et al. PLoS Genetics
- 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
- SMN Deficiency Causes Tissue-Specific Perturbations in the Repertoire of snRNAs and Widespread Defects in Splicing
- (2008) Zhenxi Zhang et al. CELL
- 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
- Embryonic motor axon development in the severe SMA mouse
- (2008) V. L. McGovern et al. HUMAN MOLECULAR GENETICS
- Regulation of SMN Protein Stability
- (2008) B. G. Burnett et al. MOLECULAR AND CELLULAR BIOLOGY
- Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
- (2008) Kevin D Foust et al. NATURE BIOTECHNOLOGY
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