Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials.
about
Is spinal muscular atrophy a disease of the motor neurons only: pathogenesis and therapeutic implications?Synthetic biology and therapeutic strategies for the degenerating brain: Synthetic biology approaches can transform classical cell and gene therapies, to provide new cures for neurodegenerative diseasesGene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model.RNA therapeutics: RNAi and antisense mechanisms and clinical applicationsSpinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.A future perspective on neurodegenerative diseases: nasopharyngeal and gut microbiota.A qualitative study of perceptions of meaningful change in spinal muscular atrophySerotonin and Histamine Therapy Increases Tetanic Forces of Myoblasts, Reduces Muscle Injury, and Improves Grip Strength Performance of Dmd(mdx) Mice.LIGAND-RECEPTOR INTERACTIONS AND DRUG DESIGN.The delivery of therapeutic oligonucleotidesMorpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics.Survival benefit and phenotypic improvement by hamartin gene therapy in a tuberous sclerosis mouse brain model.New developments in exon skipping and splice modulation therapies for neuromuscular diseases.Cystic fibrosis newborn screening: a model for neuromuscular disease screening?Splicing modulation therapy in the treatment of genetic diseases.Review of Spinal Muscular Atrophy (SMA) for Prenatal and Pediatric Genetic Counselors.Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy.Translocator protein (TSPO) ligands for the diagnosis or treatment of neurodegenerative diseases: a patent review (2010 - 2015; part 2).A Review on Spinal Muscular Atrophy: Awareness, Knowledge, and Attitudes.Pharmacomodulation of microRNA Expression in Neurocognitive Diseases: Obstacles and Future Opportunities.Therapeutic targeting of splicing in cancer.Translocator protein (TSPO) ligands for the diagnosis or treatment of neurodegenerative diseases: a patent review (2010-2015; part 1).Calpain research for drug discovery: challenges and potential.Modulation of aberrant splicing in human RNA diseases by chemical compounds.Gestational Age-Dependent Increase of Survival Motor Neuron Protein in Umbilical Cord-Derived Mesenchymal Stem Cells.Treatment Advances in Spinal Muscular Atrophy.Alternative Splicing in Breast Cancer and the Potential Development of Therapeutic Tools.The RD-Connect Registry & Biobank Finder: a tool for sharing aggregated data and metadata among rare disease researchers.Quantitative muscle ultrasound measures rapid declines over time in children with SMA type 1.
P2860
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P2860
Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials.
description
2013 nî lūn-bûn
@nan
2013 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2013 թվականի դեկտեմբերին հրատարակված գիտական հոդված
@hy
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
name
Molecular therapeutic strategi ...... nt and future clinical trials.
@ast
Molecular therapeutic strategi ...... nt and future clinical trials.
@en
Molecular therapeutic strategi ...... nt and future clinical trials.
@nl
type
label
Molecular therapeutic strategi ...... nt and future clinical trials.
@ast
Molecular therapeutic strategi ...... nt and future clinical trials.
@en
Molecular therapeutic strategi ...... nt and future clinical trials.
@nl
prefLabel
Molecular therapeutic strategi ...... nt and future clinical trials.
@ast
Molecular therapeutic strategi ...... nt and future clinical trials.
@en
Molecular therapeutic strategi ...... nt and future clinical trials.
@nl
P2093
P50
P1476
Molecular therapeutic strategi ...... ent and future clinical trials
@en
P2093
Chiara Simone
Chiara Zanetta
Erika Monguzzi
P304
P356
10.1016/J.CLINTHERA.2013.11.006
P577
2013-12-17T00:00:00Z