Cellular and animal models for facioscapulohumeral muscular dystrophy
DeSimone AM, Cohen J, Lek M, Lek A. Cellular and animal models for facioscapulohumeral muscular dystrophy. Disease Models & Mechanisms 2020, 13: dmm046904. PMID: 33174531, PMCID: PMC7648604, DOI: 10.1242/dmm.046904.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsFacioscapulohumeral muscular dystrophyMicrosatellite repeat arraysNon-primate animalsRepeat arrayFSHD pathologyCellular pathwaysChromosome 4Myogenic differentiationExpression patternsMosaic expression patternMuscular dystrophyNew disease modelsHuman diseasesCellular modelMolecular factorsHumeral musclesSkeletal muscleDisease modelsMisexpressionDUX4GenesDrug candidatesTransgenicDifferentiationPathway