Spinal muscular atrophy--recent therapeutic advances for an old challenge.
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Molecular Mechanisms of Neurodegeneration in Spinal Muscular AtrophyFrom gene discovery to new biological mechanisms: heparanases and congenital urinary bladder diseaseSpinal Muscular Atrophy: From Defective Chaperoning of snRNP Assembly to Neuromuscular Dysfunction.An Integrative Transcriptomic Analysis for Identifying Novel Target Genes Corresponding to Severity Spectrum in Spinal Muscular AtrophySmall Molecules in Development for the Treatment of Spinal Muscular Atrophy.Activin Receptor Type IIB Inhibition Improves Muscle Phenotype and Function in a Mouse Model of Spinal Muscular AtrophymicroRNA-33 Regulates ApoE Lipidation and Amyloid-β Metabolism in the BrainAMPA GluA1-flip targeted oligonucleotide therapy reduces neonatal seizures and hyperexcitabilityDisease burden of spinal muscular atrophy in Germany.Cross-disease comparison of amyotrophic lateral sclerosis and spinal muscular atrophy reveals conservation of selective vulnerability but differential neuromuscular junction pathology.Development of Therapeutics for C9ORF72 ALS/FTD-Related Disorders.RNA mis-splicing in disease.Longitudinal characterization of biomarkers for spinal muscular atrophy.A Review on Spinal Muscular Atrophy: Awareness, Knowledge, and Attitudes.Non-coding RNAs as drug targets.Post-modern therapeutic approaches for progressive myoclonus epilepsy.Diverse role of survival motor neuron protein.Motor neuron mitochondrial dysfunction in spinal muscular atrophyEfficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders.Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.Clinical Characteristics of Spinal Muscular Atrophy in Korea Confirmed by Genetic AnalysisThree-Dimensional Organoid System Transplantation Technologies in Future Treatment of Central Nervous System Diseases.Genome engineering: a new approach to gene therapy for neuromuscular disorders.Genotype-Phenotype Correlation of SMN1 and NAIP Deletions in Korean Patients with Spinal Muscular Atrophy.Survival Motor Neuron Protein is Released from Cells in Exosomes: A Potential Biomarker for Spinal Muscular Atrophy.How far away is spinal muscular atrophy gene therapy?MicroRNAs in brain cholesterol metabolism and their implications for Alzheimer's disease.Time Is Motor Neuron: Therapeutic Window and Its Correlation with Pathogenetic Mechanisms in Spinal Muscular Atrophy.miRNA in spinal muscular atrophy pathogenesis and therapy.Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape.Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs.Advances in spinal muscular atrophy therapeutics.Counting the cost of spinal muscular atrophy.Discovery of Tirasemtiv, the First Direct Fast Skeletal Muscle Troponin Activator.Dissecting the Causal Mechanism of X-Linked Dystonia-Parkinsonism by Integrating Genome and Transcriptome Assembly.Spinal muscular atrophy type I and the dual role of viruses: An interview with Professor Basil T. Darras, Professor of Neurology (Pediatrics) at Harvard Medical School.[Deregulation of pre-messenger RNA splicing and rare diseases].Recent Advances in Antisense Oligonucleotide Therapy in Genetic Neuromuscular Diseases.Blocking p62-dependent SMN degradation ameliorates spinal muscular atrophy disease phenotypes
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Spinal muscular atrophy--recent therapeutic advances for an old challenge.
description
2015 nî lūn-bûn
@nan
2015年の論文
@ja
2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
2015年论文
@zh
2015年论文
@zh-cn
name
Spinal muscular atrophy--recent therapeutic advances for an old challenge.
@en
type
label
Spinal muscular atrophy--recent therapeutic advances for an old challenge.
@en
prefLabel
Spinal muscular atrophy--recent therapeutic advances for an old challenge.
@en
P2093
P2860
P50
P356
P1476
Spinal muscular atrophy--recent therapeutic advances for an old challenge
@en
P2093
Giacomo P Comi
Irene Faravelli
Monica Nizzardo
Stefania Corti
P2860
P2888
P304
P356
10.1038/NRNEUROL.2015.77
P407
P577
2015-05-19T00:00:00Z
P6179
1039336144