Myotonic Dystrophy (DM) is a genetic disorder that affects muscle function and other systems of the body. There are two main types: 1. Myotonic Dystrophy Type 1 (DM1): Also known as Steinert's disease, DM1 is caused by a mutation in the "DMPK gene" (Dystrophia Myotonica Protein Kinase). The mutation involves an expansion of CTG trinucleotide repeats in this gene, leading to symptoms such as prolonged muscle contractions (myotonia), muscle weakness, …

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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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