The main focus of Dr. Gomez-Ospina’s lab is to develop therapies for patients with genetic neurodgenerative diseases. The lab uses genome editing and stem cells to produce definitive treatments for childhood neurodegenerative diseases, many of which are lysosomal storage disorders.
Current projects in the lab include developing autologous transplantation of genome-edited hematopoietic stem cells for Mucopolysaccharidosis type I, Gaucher, Krabbe disease, Frontotemporal Dementia, and Friedreich's ataxia.
Although there is a strong translational focus to the lab, we are also pursuing basic science questions to understand and enhance our therapies including: 1) increasing the efficiency of genome editing tools, 2) understanding microglia turnover in response to conditioning before hematopoietic stem transplant, and 3) stablishing brain-specific conditioning regimens to neurometabolic diseases.