Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds

Fasudil improves survival and promotes skeletal muscle development in a mouse model of spinal muscular atrophy

(2012) Melissa Bowerman et al.   BMC Medicine

SMN-inducing compounds for the treatment of spinal muscular atrophy

(2012) Monique A Lorson et al.   Future Medicinal Chemistry

Therapeutic strategies for the treatment of spinal muscular atrophy

(2012) Jonathan J Cherry et al.   Future Medicinal Chemistry

Identification of a Maleimide-Based Glycogen Synthase Kinase-3 (GSK-3) Inhibitor, BIP-135, That Prolongs the Median Survival Time of Δ7 SMA KO Mouse Model of Spinal Muscular Atrophy

(2011) Po C. Chen et al.   ACS Chemical Neuroscience

Increasing expression and decreasing degradation of SMN ameliorate the spinal muscular atrophy phenotype in mice

(2011) Deborah Y. Kwon et al.   HUMAN MOLECULAR GENETICS

A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

(2011) Paul N. Porensky et al.   HUMAN MOLECULAR GENETICS

Synthesis and Characterization of Pseudocantharidins, Novel Phosphatase Modulators That Promote the Inclusion of Exon 7 into theSMN(Survival of Motoneuron) pre-mRNA

(2011) Zhaiyi Zhang et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

Discovery, Synthesis, and Biological Evaluation of Novel SMN Protein Modulators

(2011) Jingbo Xiao et al.   JOURNAL OF MEDICINAL CHEMISTRY

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

(2011) Yimin Hua et al.   NATURE

A screen for regulators of survival of motor neuron protein levels

(2011) Nina R Makhortova et al.   Nature Chemical Biology

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

(2011) M. A. Passini et al.   Science Translational Medicine

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

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

(2010) Elisa Dominguez et al.   HUMAN MOLECULAR GENETICS

Rho-kinase inactivation prolongs survival of an intermediate SMA mouse model

(2010) Mélissa Bowerman 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

Neuromuscular defects and breathing disorders in a new mouse model of spinal muscular atrophy

(2010) Magali Michaud et al.   NEUROBIOLOGY OF DISEASE

Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy

(2010) C. F. Valori et al.   Science Translational Medicine

Therapeutic developments in spinal muscular atrophy

(2010) Douglas M. Sproule et al.   Therapeutic Advances in Neurological Disorders

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

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

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

p38 Mitogen-activated protein kinase stabilizes SMN mRNA through RNA binding protein HuR

(2009) Faraz Farooq 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

Tetracyclines That Promote SMN2 Exon 7 Splicing as Therapeutics for Spinal Muscular Atrophy

(2009) M. L. Hastings et al.   Science Translational Medicine

DcpS as a Therapeutic Target for Spinal Muscular Atrophy

(2008) Jasbir Singh et al.   ACS Chemical Biology

Sustained improvement of spinal muscular atrophy mice treated with trichostatin a plus nutrition

(2008) Heather L. Narver et al.   ANNALS OF NEUROLOGY

Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation

(2008) Kathrin Meyer et al.   HUMAN MOLECULAR GENETICS

Embryonic motor axon development in the severe SMA mouse

(2008) V. L. McGovern et al.   HUMAN MOLECULAR GENETICS

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

Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: Applicability to preclinical therapy development for spinal muscular atrophy

(2008) Virginia B. Mattis et al.   JOURNAL OF NEUROSCIENCE METHODS

Regulation of SMN Protein Stability

(2008) B. G. Burnett et al.   MOLECULAR AND CELLULAR BIOLOGY

Development of a Single Vector System that Enhances Trans-Splicing of SMN2 Transcripts

(2008) Tristan H. Coady et al.   PLoS One