Targeted epigenetic repression by CRISPR/dSaCas9 suppresses pathogenic DUX4-fl expression in FSHD
Charis L. Himeda, Takako I. Jones, Peter L. Jones
Abstract
delivery. By engineering a muscle-specific epigenetic CRISPR platform compatible with AAV vectors for gene therapy, we have laid the groundwork for clinical use of dCas9-based chromatin effectors in skeletal muscle disorders.
Topics & Concepts
Facioscapulohumeral muscular dystrophyBiologyCRISPRChromatinSkeletal muscleEpigeneticsPsychological repressionGene silencingMuscle disorderGeneticsCell biologyMuscular dystrophyGeneGene expressionMedicineAnatomyInternal medicineCRISPR and Genetic EngineeringVirus-based gene therapy researchMuscle Physiology and Disorders