1. Introduction: In the last 25 years, 18 oligonucleotide therapeutics have been approved for treating diseases like spinal muscular atrophy, Duchenne muscular dystrophy, and hereditary transthyretin-mediated amyloidosis. These therapies operate through different mechanisms, including antisense oligonucleotides (ASOs), splice-switching oligonucleotides (SSOs), RNA interference (RNAi), and RNA aptamers. 2. Historical Background: Oligonucleotide therapeutics trace back to the 1950s and 1960s with early discoveries in DNA and RNA synthesis. Pioneers like Todd, Khorana, …

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  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 …

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