Patients with Duchenne muscular dystrophy (DMD) lack a functional structural protein called dystrophin in their muscle cells. However, muscle cells contain a gene for a homologous protein called utrophin, which can functionally compensate for the lack of dystrophin but is normally switched off shortly after birth.
A regulatory gene called UPR-1 produces a repressor protein that actively blocks the transcription of the utrophin gene in adult muscle tissue.
How could a therapeutic treatment targeting the UPR-1 pathway be used to treat patients with DMD?
Genetically engineering muscle cells to introduce multiple extra copies of the active UPR-1 gene.
Preventing the expression of the UPR-1 gene, allowing transcription of the utrophin gene to proceed.
Upregulating the translation of UPR-1 mRNA in the cytoplasm to increase the concentration of the repressor protein.
Deactivating all ribosomes in the skeletal muscle cells to completely stop UPR-1 protein synthesis.