Therapeutic strategies for the treatment of spinal muscular atrophy

Deacetylase Activity Is Required for STAT5-Dependent GM-CSF Functional Activity in Macrophages and Differentiation to Dendritic Cells

(2014) Carlos Sebastián et al.   JOURNAL OF IMMUNOLOGY

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

(2012) Melissa Bowerman et al.   BMC Medicine

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

Differential regulation of the SMN2 gene by individual HDAC proteins

(2011) Matthew C. Evans et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Evaluation of muscle strength and motor abilities in children with type II and III spinal muscle atrophy treated with valproic acid

(2011) Illora A Darbar et al.   BMC Neurology

Valproic acid increases SMN2 expression and modulates SF2/ASF and hnRNPA1 expression in SMA fibroblast cell lines

(2011) Indra Sari Kusuma Harahap et al.   BRAIN & DEVELOPMENT

Regulation of protein stability by GSK3 mediated phosphorylation

(2011) Chong Xu et al.   CELL CYCLE

Beta-lactam antibiotic offers neuroprotection in a spinal muscular atrophy model by multiple mechanisms

(2011) Monica Nizzardo et al.   EXPERIMENTAL NEUROLOGY

The COPI vesicle complex binds and moves with survival motor neuron within axons

(2011) Cyril Jayakumar Peter et al.   HUMAN MOLECULAR GENETICS

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

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

Utility of Survival Motor Neuron ELISA for Spinal Muscular Atrophy Clinical and Preclinical Analyses

(2011) Dione T. Kobayashi et al.   PLoS One

SMA CARNI-VAL TRIAL PART II: A Prospective, Single-Armed Trial of L-Carnitine and Valproic Acid in Ambulatory Children with Spinal Muscular Atrophy

(2011) John T. Kissel et al.   PLoS One

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

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

SMN in spinal muscular atrophy and snRNP biogenesis

(2011) Tristan H. Coady et al.   Wiley Interdisciplinary Reviews-RNA

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

Salbutamol increases survival motor neuron (SMN) transcript levels in leucocytes of spinal muscular atrophy (SMA) patients: relevance for clinical trial design

(2010) F. D. Tiziano et al.   JOURNAL OF MEDICAL GENETICS

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

Hydroxyurea enhances SMN2 gene expression through nitric oxide release

(2010) Cheng Xu et al.   NEUROGENETICS

Randomized, double-blind, placebo-controlled trial of hydroxyurea in spinal muscular atrophy

(2010) T.- H. Chen et al.   NEUROLOGY

Histological and Functional Benefit Following Transplantation of Motor Neuron Progenitors to the Injured Rat Spinal Cord

(2010) Sharyn L. Rossi et al.   PLoS One

SMA CARNI-VAL Trial Part I: Double-Blind, Randomized, Placebo-Controlled Trial of L-Carnitine and Valproic Acid in Spinal Muscular Atrophy

(2010) Kathryn J. Swoboda et al.   PLoS One

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

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

Molecular and phenotypic reassessment of an infrequently used mouse model for spinal muscular atrophy

(2009) Rocky G. Gogliotti et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA

(2009) Virginia B Mattis et al.   BMC NEUROSCIENCE

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

Induction of Nrf2 and xCT are involved in the action of the neuroprotective antibiotic ceftriaxonein vitro

(2009) Jan Lewerenz et al.   JOURNAL OF NEUROCHEMISTRY

Valproic acid blocks excitability in SMA type I mouse motor neurons

(2009) Kristen Rak et al.   NEUROBIOLOGY OF DISEASE

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

Donor-Derived Brain Tumor Following Neural Stem Cell Transplantation in an Ataxia Telangiectasia Patient

(2009) Ninette Amariglio et al.   PLOS MEDICINE

DcpS as a Therapeutic Target for Spinal Muscular Atrophy

(2008) Jasbir Singh et al.   ACS Chemical Biology

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

Riluzole in psychiatry: a systematic review of the literature

(2008) Carlos A Zarate et al.   Expert Opinion on Drug Metabolism & Toxicology

Trichostatin A causes p53 to switch oxidative-damaged colorectal cancer cells from cell cycle arrest into apoptosis

(2008) C. Habold et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

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

Synthesis and Biological Evaluation of Novel 2,4-Diaminoquinazoline Derivatives asSMN2Promoter Activators for the Potential Treatment of Spinal Muscular Atrophy

(2008) John Thurmond et al.   JOURNAL OF MEDICINAL CHEMISTRY

Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice

(2008) Li-Kai Tsai et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

Specific Antinociceptive Activity of Cholest-4-en-3-one, Oxime (TRO19622) in Experimental Models of Painful Diabetic and Chemotherapy-Induced Neuropathy

(2008) T. Bordet et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

The effect of hydroxyurea in spinal muscular atrophy cells and patients

(2008) Wen-Chen Liang et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

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

Daily salbutamol in young patients with SMA type II

(2008) Marika Pane et al.   NEUROMUSCULAR DISORDERS

Hydroxyurea enhances SMN2 gene expression in spinal muscular atrophy cells

(2005) Susanna M. Grzeschik et al.   ANNALS OF NEUROLOGY