Overactivation of Rac1, a protein that regulates components of a cell’s skeleton, may drive early nerve damage in hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN), according to new data from a preclinical study.
The research team found that reducing Rac1 activity restored nerve cell structure and prevented nerve degeneration in an hATTR-PN mouse model. Further, in analyzing patient data, the team discovered that individuals carrying a specific mutation that ultimately suppresses Rac1 activity have a delayed symptom onset.