Опубликована: Июль 22, 2024
Facioscapulohumeral muscular dystrophy (FSHD) is an inherited myopathy, characterized by progressive and asymmetric muscle atrophy, primarily affecting muscles of the face, shoulder girdle, upper arms before lower extremities with age greater disease severity. FSHD a disabling condition, patients may also present various extramuscular symptoms. caused aberrant expression double homeobox 4 (DUX4) in adult skeletal muscle, arising from compromised epigenetic repression D4Z4 array. DUX4 encodes protein, transcription factor that activates myotoxic gene programs to produce pathology. Therefore, sequence-specific oligonucleotides aimed at reducing levels compelling therapeutic approach, one has received considerable research interest over last decade. This review aims describe current preclinical landscape oligonucleotide therapies for FSHD. includes outlining mechanism action each therapy summarizing results obtained regarding their efficacy cellular and/or murine models. The scope this limited oligonucleotide-based inhibit gene, mRNA, or protein way does not involve editing.
Язык: Английский