Using cutting-edge technologies and expertise to develop and disseminate new, precise animal models of incurable and genetically complex human diseases.
Four years ago as part of The Burgess Lab at The Jackson Laboratory, Kathy Morelli and a large team of collaborators took on the challenge of creating a cure for Caroline, a young girl who was battling an incurable disease called Charcot-Marie-Tooth.
JAX Professor Robert Burgess and collaborators are developing personalized gene therapy for a Texas child suffering from a neuromuscular disease.
The 2019 Spring Report from the Charcot-Marie-Tooth Association (CMTA) highlights work being done by JAX Professor Robert Burgess and collaborators toward a clinical trial for a young girl who has CMT type 2D, a rare and severe form of the disease.
For the rarest of rare genetic conditions, a one-of-a-kind mouse could light a path to new treatments.