Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
出版年份 2011 全文链接
标题
Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
作者
关键词
-
出版物
Science Translational Medicine
Volume 3, Issue 72, Pages 72ra18-72ra18
出版商
American Association for the Advancement of Science (AAAS)
发表日期
2011-03-04
DOI
10.1126/scitranslmed.3001777
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform
- (2010) C. Frank Bennett et al. Annual Review of Pharmacology and Toxicology
- Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
- (2010) Y. Hua et al. GENES & DEVELOPMENT
- SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy
- (2010) Markus Riessland 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
- Spinal muscular atrophy: mechanisms and therapeutic strategies
- (2010) C. L. Lorson 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
- Trans-Splicing-Mediated Improvement in a Severe Mouse Model of Spinal Muscular Atrophy
- (2010) T. H. Coady et al. JOURNAL OF NEUROSCIENCE
- 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 short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy
- (2010) Natalia N. Singh et al. RNA Biology
- Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy
- (2010) C. F. Valori et al. Science Translational Medicine
- Embryonic stem cell-derived neural stem cells improve spinal muscular atrophy phenotype in mice
- (2009) Stefania Corti et al. BRAIN
- Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model
- (2009) Virginia B. Mattis et al. HUMAN MOLECULAR GENETICS
- 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
- LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate
- (2009) Lutz Garbes 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
- 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
- Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons
- (2009) Sandra Duque et al. MOLECULAR THERAPY
- Phase II Open Label Study of Valproic Acid in Spinal Muscular Atrophy
- (2009) Kathryn J. Swoboda et al. PLoS One
- Tetracyclines That Promote SMN2 Exon 7 Splicing as Therapeutics for Spinal Muscular Atrophy
- (2009) M. L. Hastings et al. Science Translational Medicine
- 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
- Sustained improvement of spinal muscular atrophy mice treated with trichostatin a plus nutrition
- (2008) Heather L. Narver et al. ANNALS OF NEUROLOGY
- Neural stem cell transplantation can ameliorate the phenotype of a mouse model of spinal muscular atrophy
- (2008) Stefania Corti et al. JOURNAL OF CLINICAL INVESTIGATION
- Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice
- (2008) Li-Kai Tsai et al. JOURNAL OF MOLECULAR MEDICINE-JMM
- Spinal muscular atrophy
- (2008) Maryam Oskoui et al. Neurotherapeutics
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExplorePublish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn More