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Oligonucleotide Therapies for Facioscapulohumeral Muscular Dystrophy: Current Preclinical Landscape
Personal take on this article: Facioscapulohumeral muscular dystrophy (FSHD) is a progressive genetic disorder that primarily affects muscles of the face, shoulder girdle, and upper arms, with the potential to cause severe muscle atrophy over time. The disease is characterized by the aberrant expression of the DUX4 gene due to compromised epigenetic repression of the D4Z4 array, leading to toxic effects in muscle tissue. FSHD can be categorized into …