Mark Osborn, PhD

Dr. Osborn's current research is focused on gene and cellular therapy for disorders treated by hematopoietic cell transplantation including: Fanconi anemia, epidermolysis bullosa, and Hurler syndrome. This involves utilizing a patient's own cells for precision gene targeting and correction of their disease causing mutation. Genome editing nucleases and multiple terminally differentiated and stem cell populations are utilized towards optimizing ex vivo cellular therapies.
Gene Therapy
- Non-viral gene transfer for the treatment of inborn errors of metabolism.
Genome Editing
- Correction of disease specific mutations in a precise manner using homologous recombination.
Cellular Therapy
- Ex vivo correction of murine and human adult stem cells.