University of Pennsylvania · NeuroscienceBennett Cell Lab
Frederick Bennett · Philadelphia, Pennsylvania
Research Focus
Dr. Frederick Bennett's lab at the University of Pennsylvania focuses on developing innovative cell therapies for neurodegenerative diseases, particularly leukodystrophies like Krabbe disease. The research aims to improve the efficacy of hematopoietic stem cell transplantation by engineering immune cells that can effectively replace damaged brain cells. By optimizing these techniques in animal models, the lab is paving the way for potential new treatments to help young patients suffering from this devastating condition.
Krabbe diseaseleukodystrophiesmicroglial replacementstem cell therapygene editingCNS engraftmentHSC transplantationpreclinical models
Research Aims
3AIM 01Validate a method to engineer murine HSCs for effective engraftment using a CSF1R inhibitor resistant variant.
AIM 02Demonstrate the engraftment and effects of genetically modified HSCs in a mouse model of Krabbe disease.
AIM 03Establish the safety and efficacy of HSC replacement in nonhuman primates to support clinical translation.
Skills the Lab Values
mouse modelsgene editingcell transplantationin vivo imaginggene therapy techniquescell culturebiochemical assaysintracranial deliveryCSF1R inhibition