University of Southern California · Microbiology & ImmunologyRNA Therapeutics and Drug Discovery Lab
Lucio Comai · Los Angeles, California
Research Focus
Dr. Lucio Comai's research lab focuses on discovering new small molecule drugs for treating myotonic dystrophy type 1 (DM1), a serious genetic disorder. The lab employs innovative RNA-based screening methods to identify compounds that can effectively target and modulate disease-causing RNA, ultimately aiming to improve the lives of patients suffering from this debilitating condition. By leveraging advanced techniques and a diverse library of potential drugs, the lab is making strides in finding effective therapies where few options currently exist.
myotonic dystrophysmall molecule therapeuticsRNA modulationdrug discoverymuscle disease
Research Aims
5AIM 01Identify small molecules that selectively target CUGexp RNA in DM1.
AIM 02Evaluate the efficacy and safety of novel drug compounds.
AIM 03Understand the mechanism of action of top-ranked drug candidates.
AIM 04Test drug candidates for their ability to reverse DM1 pathology in mice.
AIM 05Assess blood-brain barrier penetrance of new therapeutic hits.
Skills the Lab Values
RNA-based screeningbiochemical assaysmouse modelscell culturedrug library screening
Output against the department
Publications275top 19% of dept.91st of 486
this lab
most publishedleast published
Citations4.5kbottom 35% of dept.317th of 486
this lab
most citedleast cited
Career totals from OpenAlex, compared against labs that share a department. A department spans everything from one paper to tens of thousands, so bar heights are on a log scale — otherwise the largest lab would flatten every other bar to a line.