Bringing together global mouse resources for a start-to-finish approach to model development.
By definition, a rare and orphan disease is one that affects fewer than 200,000 individuals in the United States. Because of the small population afflicted by any one illness, funding to investigate causes and treatments tends to be limited, slowing the discovery of potential therapies. Yet with over 7,000 recognized rare diseases, an estimated 350 million people worldwide are affected at any given time.
The Rare and Orphan Disease Center brings together global mouse resources and the expertise of scientists at the Jackson Laboratory for a start-to-finish approach to model development. A cure for any disease will not happen without concerted effort from people around the world. We partner with foundations, pharmaceutical and biotech companies, and other scientists worldwide to facilitate research into treatments of these less common diseases.
JAX Professor Robert Burgess and collaborators are developing personalized gene therapy for a Texas child suffering from a neuromuscular disease.
Cortney Lavorgna is a college student who is exploring the neuromuscular disease she lives with in a JAX research experience.
Neuromuscular diseases start by hampering mobility and progress to threatening vital processes like breathing and swallowing. Greg Cox wants to knock them all out.
For the rarest of rare genetic conditions, a one-of-a-kind mouse could light a path to new treatments.