Article
Transcriptional adaptation upregulates utrophin in Duchenne muscular dystrophy.
Nature - 1 Mar 2025
Falcucci Lara, Dooley Christopher M, Adamoski Douglas, Juan Thomas, Martinez Justin, Georgieva Angelina M, Mamchaoui Kamel, Cirzi Cansu, Stainier Didier Y R
Abstract excerpt
Duchenne muscular dystrophy (DMD) is a muscle-degenerating disease caused by mutations in the DMD gene, which encodes the dystrophin protein1,2. Utrophin (UTRN), the genetic and functional paralogue of DMD, is upregulated in some DMD patients3-5. To further investigate this UTRN upregulation, we first developed an inducible messenger RNA (mRNA) degradation system for DMD by introducing a premature termination...
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