Understanding Neuromuscular Health and Disease: Advances in Genetics, Omics, and Molecular Function

Understanding Neuromuscular Health and Disease: Advances in Genetics, Omics, and Molecular Function

This compilation focuses on recent advances in the molecular and cellular understandingof neuromuscular biology, and the treatment of neuromuscular disease.These advances are at the forefront of modern molecular methodologies, oftenintegrating across wet-lab cell and tissue models, dry-lab computati...

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Bibliographic Details
Format:	eBook
Language:	English
Published:	Basel, Switzerland MDPI - Multidisciplinary Digital Publishing Institute 2021
Subjects:	Research & information: general -omics approaches AAV adult patients ALS ALS genes ALS pathology ALS variants Amyotrophic Lateral Sclerosis autophagy axonal transport Becker muscular dystrophy becker muscular dystrophy (BMD) biomarkers calprotectin Canadian Neuromuscular Disease Registry clinical trials comparative genomic hybridization array (CGH) corticosteroids CRISPR-Cas9 D4Z4 deflazacort disease heterogeneity disease models disease modifiers DMARD DMD Duchenne muscular dystrophy duchenne muscular dystrophy (DMD) Duchenne muscular dystrophy (DMD) DUX4 dystrophin dystrophinopathy dystrophy Emery-Dreifuss muscular dystrophy endocytosis epigenetic changes eteplirsen excitotoxicity exon skipping exon skipping therapy exon-skipping therapies facioscapulohumeral dystrophy FSHD functional outcomes gene editing gene prioritization gene therapy genetic neuromuscular disorders genome-wide association studies genomics genotype-phenotype genotype-phenotype correlations glucocorticoids golodirsen GWAS LMNA machine learning methotrexate miRNA mitochondria dysfunction MND multiple logistic regression analysis multiplex ligation probe amplification (MLPA) multiplex polymerase chain reaction (PCR) muscle n/a neuromuscular diseases next-generation sequencing (NGS) Nusinersen Omics oxidative stress pharmacodynamic biomarkers pharmacogenomics polyadenylation precision medicine prednisone proteomics reading frame rule regulation rheumatoid arthritis RNA metabolism safety Sanger sequencing secretion skip-equivalent deletions SMA SNP spinal muscular atrophy TALEN toxicity transcription translational research viltolarsen XLMTM
Online Access:	Open Access: DOAB: description of the publication Open Access: DOAB, download the publication

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