Professor of Pediatrics and 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 Program Director
UT Southwestern Medical Center
What first drew you to Krabbe disease—and why have you stayed?
I got pulled into this field by colleagues who thought the gene therapy technology I was working on could be helpful. I’ve stayed in it because the gene therapy approaches, we’ve developed look very promising and I’d like to see this technology help Krabbe patients.
How do patient families influence or motivate your work?
They are the reason I do this work in Krabbe disease—and why I continue to stay involved. Patients need better treatments.
What keeps you going on the hard days when progress feels slow?
The patients need help. We need to deliver new treatments to them.
