The article delves into Duchenne muscular dystrophy (DMD), a genetic disorder leading to progressive muscle degeneration due to dystrophin gene mutations. Current FDA-approved treatments include exon skipping and gene therapies. Research suggests that utrophin, a dystrophin homolog, is upregulated in patients with certain DMD mutations, which may protect against the disease's severity. The study investigates how DMD mutant mRNA decay impacts utrophin levels, proposing a concept termed transcriptional adaptation that may pave the way for new therapeutic strategies targeting utrophin upregulation in DMD.
In our study, we uncover that messenger RNA decay of mutated DMD mRNA triggers a compensatory increase in UTRN, driven by a process we term transcriptional adaptation.
Our findings highlight the significance of transcriptional adaptation in the regulation of UTRN expression, providing insights into the molecular mechanisms that could be targeted for DMD therapies.
Collection
[
|
...
]