Natalia Gomez-Ospina
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.