This paper investigates a potential treatment for facioscapulohumeral muscular dystrophy (FSHD), a genetic disorder caused by the improper expression of the DUX4 gene in muscles, leading to muscle weakness and deterioration. Currently, there is no molecular therapy to halt or slow the progression of FSHD. The researchers tested an antisense oligonucleotide (ASO) that targets and reduces the DUX4 transcript in a mouse model (ACTA1-MCM;FLExDUX4) that mimics FSHD. The ASO treatment, …

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Personal take on this article: This paper discusses the work of the Dutch Center for RNA Therapeutics (DCRT) in developing individualized antisense oligonucleotides (ASOs) for patients with rare brain or eye diseases. ASOs are short pieces of modified DNA that can adjust gene expression and have the potential to treat genetic disorders. However, many rare diseases are specific to individual patients, meaning pharmaceutical companies often overlook these cases due to …

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