Jerome Harris Distinguished Professor of Pediatrics
Professor of Pathology
Director, Marcus Center for Cellular Cures
Director, Carolinas Cord Blood Bank
Co-Director, Stem Cell TransplantTypically this refers to a bone marrow transplant with the goal of replacing non-working cells with healthy working cells. Bone marrow is a rich source of stem cells that have the unique ability to Laboratory
President, Cord Blood Association
What is one thing you wish every family understood about the work happening right now?
Two things: (1) While progress is being made, it’s moving more slowly than we would like. We have to give it more time than we planned, but we will get there; (2) If you know or are trying to help a family with a potential newly diagnosed individual, get them to the experts who can interpret their results and give them advice about next steps. Krabbe disease diagnosis can be complicated. With newborn screening, we are learning new things every day. It’s important to talk with healthcare professionals who know about Krabbe disease to help families make their best choices for treatment and or follow-up.
What gives you the most hope when you think about the future of Krabbe disease?
I believe we will cure Krabbe Disease using multimodal therapy. Currently, gene therapyA type of therapy that offers hope and promise for a cure for many genetic disorders. A working copy of the gene replaces the non-working copy of the gene. Gene therapy is at the forefront of many is the most promising next advancement, but there are many different approaches and methods of delivery being explored. We still have a lot to learn about which type(s) is/are best, what route to administer it, and which phases of Krabbe disease can help. We also need more research into other types of treatments to augment transplants and gene therapy.
What’s something about you outside the lab that might surprise families?
I knit and I have a poorly behaved but very cute French bulldog.
How do patient families influence or motivate your work?
I am continuously amazed by the resilience of families dealing with Krabbe disease. They are a constant reminder of how to prioritize the important things in life.
