Project description:Spinal Muscular Atrophy (SMA) is an autosomal recessive motor neuron disease and is the second most common genetic disorder leading to death in childhood. Stem cell transplantation could represent a therapeutic approach for motor neuron diseases such as SMA. We examined the theraputics effects of a spinal cord neural stem cell population and their ability to modify SMA phenotype. Microarray technology was used to assess the global gene expression profile of laser-microdissected motoneurons obtained by transplanted and veichle treated SMA, and wildtype mice. Keywords: Comparative Gene Expression Analysis
Project description:Spinal Muscular Atrophy (SMA) is an autosomal recessive motor neuron disease and is the second most common genetic disorder leading to death in childhood. Stem cell transplantation could represent a therapeutic approach for motor neuron diseases such as SMA. We examined the theraputics effects of a spinal cord neural stem cell population and their ability to modify SMA phenotype. Microarray technology was used to assess the global gene expression profile of laser-microdissected motoneurons obtained by transplanted and veichle treated SMA, and wildtype mice. Experiment Overall Design: The microarray data derived from three different groups: wildtype controls (vehicle treated), transgenic SMA (vehicle treated) and transplanted SMA mice. Each population consists of three RNA profiling samples.
Project description:Study of gene expression profiles of muscular and neuronal mouse mutant of spinal muscular atrophy(SMA). Pre and post symptomatic stage disease have been analyzed.
Project description:We here longitudinally investigated how spinal muscular atrophy (SMA) and nusinersen shaped local immune responses in the cerebrospinal fluid (CSF).
Project description:Spinal Muscular Atrophy (SMA) is an autosomal recessive motor neuron disease and is the second most common genetic disorder leading to death in childhood. No effective therapy is currently available. It has been suggested that M-NM-2-lactam antibiotics such as ceftriaxone may offer neuroprotection in motoneuron disease. We investigated the therapeutic effect of ceftriaxone in a murine model of SMA. Microarray technology was used to assess the global gene expression profile of spinal cord obtained by ceftriaxone-treated and vehicle treated SMA mice. Comparative Gene Expression Analysis. The microarray data derived from three different groups: wildtype controls, transgenic SMA (vehicle treated) and ceftriaxone-treated SMA mice. Each population consists of four RNA profiling samples.
Project description:Selective motor neuron degeneration in spinal muscular atrophy (SMA) is driven by converging mechanisms that promote stabilization and phosphorylation of p53 in vulnerable motor neurons. However, the downstream transcriptional programs that execute motor neuron death remain incompletely defined.To characterize p53-associated transcriptional changes in vulnerable motor neurons, we performed RNA sequencing of cholera toxin subunit B (CTB)-labeled lumbar (L1–L3) motor neurons isolated by laser-capture microdissection from postnatal day 6 SMNΔ7 (SMNΔ7;SMN2;Smn−/−) SMA mice and wild-type littermate controls. Disease-associated gene expression changes were defined by comparison of SMA and wild-type motor neurons. To assess pathway-specific contributions, untreated SMA mice were compared to SMA mice treated with Pifithrin-α (inhibitor of p53 transcriptional activity), MW150 (p38 MAPK inhibitor that prevents p53 phosphorylation), or SMN-C3 (SMN splicing modifier). Differential gene expression analysis was performed using kallisto-based transcript quantification and DESeq2.
Project description:In this study, label-free quantitative proteomic analysis was performed using the Smn 2B/- mouse model to identify and investigate significant changes in protein abundance that may be related to the pathogenesis and neurodegeneration oberved in spinal muscular atrophy (SMA)
Project description:Spinal Muscular Atrophy (SMA) is an autosomal recessive motor neuron disease and is the second most common genetic disorder leading to death in childhood. No effective therapy is currently available. It has been suggested that β-lactam antibiotics such as ceftriaxone may offer neuroprotection in motoneuron disease. We investigated the therapeutic effect of ceftriaxone in a murine model of SMA. Microarray technology was used to assess the global gene expression profile of spinal cord obtained by ceftriaxone-treated and vehicle treated SMA mice.
Project description:RNA-seq transcriptome profiling of motor neurons (MNs) and surrounding white matter glia (WM) microdissected from pre-symptomatic mouse model of spinal muscular atrophy (SMA)
