University of Illinois Chicago · BiologyKrabbe Disease Therapeutics Lab
Ernesto Roque Bongarzone · Chicago, Illinois
Research Focus
Dr. Ernesto Roque Bongarzone's lab at the University of Illinois at Chicago is focused on developing new therapies for Krabbe disease, a genetic disorder that affects the nervous system. The lab studies various treatment strategies including gene therapy and small molecule inhibitors that could help reduce toxic levels of psychosine, a harmful substance that accumulates in patients with this condition. By improving treatment options, this research aims to enhance the quality of life for individuals affected by Krabbe disease and potentially other related disorders.
Krabbe diseaselysosomal storage diseasesgene therapypsychosinesubstrate reduction therapy
Research Aims
5AIM 01Develop new combination therapies for Krabbe disease.
AIM 02Evaluate the efficacy of CGT and ACD inhibitors in mouse models.
AIM 03Investigate how to enhance existing treatments like HSCT and AAV gene therapy.
AIM 04Understand the disease mechanisms associated with psychosine accumulation.
AIM 05Explore treatment possibilities for juvenile and adult onset Krabbe disease.
Skills the Lab Values
mouse modelsgene therapy techniquesdrug screeningin vivo efficacy testingcell culture
Output against the department
Publications138top 50% of dept.518th of 1,033
this lab
most publishedleast published
Citations4.6kbottom 35% of dept.669th of 1,033
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.