Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model
出版年份 2009 全文链接
标题
Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model
作者
关键词
-
出版物
HUMAN MOLECULAR GENETICS
Volume 18, Issue 20, Pages 3906-3913
出版商
Oxford University Press (OUP)
发表日期
2009-07-23
DOI
10.1093/hmg/ddp333
参考文献
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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。- Translational readthrough by the aminoglycoside geneticin (G418) modulates SMN stability in vitro and improves motor function in SMA mice in vivo
- (2009) Christopher R. Heier 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
- Impaired Synaptic Vesicle Release and Immaturity of Neuromuscular Junctions in Spinal Muscular Atrophy Mice
- (2009) L. Kong et al. JOURNAL OF NEUROSCIENCE
- Sustained improvement of spinal muscular atrophy mice treated with trichostatin a plus nutrition
- (2008) Heather L. Narver et al. ANNALS OF NEUROLOGY
- SMN Deficiency Causes Tissue-Specific Perturbations in the Repertoire of snRNAs and Widespread Defects in Splicing
- (2008) Zhenxi Zhang et al. CELL
- A Negatively Acting Bifunctional RNA Increases Survival Motor Neuron Both In Vitro and In Vivo
- (2008) Alexa Dickson et al. HUMAN GENE THERAPY
- Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy
- (2008) Ferrill F. Rose et al. HUMAN MOLECULAR GENETICS
- Reduced SMN protein impairs maturation of the neuromuscular junctions in mouse models of spinal muscular atrophy
- (2008) Shingo Kariya et al. HUMAN MOLECULAR GENETICS
- Embryonic motor axon development in the severe SMA mouse
- (2008) V. L. McGovern et al. HUMAN MOLECULAR GENETICS
- SMN complex localizes to the sarcomeric Z-disc and is a proteolytic target of calpain
- (2008) Michael P. Walker et al. HUMAN MOLECULAR GENETICS
- Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial
- (2008) Eitan Kerem et al. LANCET
- Regulation of SMN Protein Stability
- (2008) B. G. Burnett et al. MOLECULAR AND CELLULAR BIOLOGY
- Induced pluripotent stem cells from a spinal muscular atrophy patient
- (2008) Allison D. Ebert et al. NATURE
- A SMNΔ7 read-through product confers functionality to the SMNΔ7 protein
- (2008) Virginia B. Mattis et al. NEUROSCIENCE LETTERS
- Development of a Single Vector System that Enhances Trans-Splicing of SMN2 Transcripts
- (2008) Tristan H. Coady et al. PLoS One
- PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model
- (2008) M. Du et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Introducing sense into nonsense in treatments of human genetic diseases
- (2008) Liat Linde et al. TRENDS IN GENETICS
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