Abstract
A new molecular mechanism of trinucleotide expansion diseases is suggested. The mechanism involves the formation of double-helical RNA hairpins by transcripts carrying (CNG)n sequences, which are processed via the RNAi pathway with subsequent RNA silencing of genes containing (CNG)n sequences. Depletion of proteins encoded by these genes leads to the specific disease phenotype. The available data on human myotonic dystrophy 1, which results from the (CTG)n expansion, support the hypothesis.
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