Organoids reveal similarities between myotonic dystrophy type 1 and Rett syndrome
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29 June 2022Organoids reveal similarities between myotonic dystrophy type 1 and Rett syndrome
Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy, characterized by progressive muscle wasting and weakness and caused by abnormally repetitive DNA segments that are transcribed into toxic molecules of RNA. Instead of ferrying a gene’s instructions for translation into proteins, these RNA molecules accumulate in cells, disrupting cellular machinery.
