Insights into the molecular pathology of SBMA: Targeting PRMT6 to attenuate the disease.
Insights into the molecular pathology of SBMA: Targeting PRMT6 to attenuate the disease.
In collaboration with the Laboratory of Dr. Pennuto, we have recently discovered that a protein, called PRMT6, exacerbates the toxicity induced by mutant androgen receptor, while its inhibition rescues it in cells and flies. Our strategy is to develop a therapy that preserves AR physiological functions while abolishing the toxicity acquired upon polyglutamine expansion. Thus, we propose to silence PRMT6 both via selective pharmacological inhibitors and via gene-silencing to choose the best system to move our studies in pre-clinical models.
Provide important information when developing new therapeutics for this devastating disease.
