12/11/2025
Here's the latest good news with Paxton...
Hello Friends, We have an exciting update to share on the gene therapy project at UMass Chan Medical School! UMass has let us know that the vector (which will be used to deliver the gene therapy) has been built! It is currently being tested in cultures to assess ongoing protein expression and silencing the endogenous gene. Next steps will be to create plasmids to then inject TFRC (Transferrin Receptor) into mice with CLCN6 mutations. Jackson Laboratory is building out the colony and we will share an update when that is made available. This is a big step in the project and we would not be here if it wasn't for the support of each and every one of you who has donated to us. There is still a long way to go, but we do want to take a moment to celebrate this milestone and express our gratitude and appreciation to each and every person who has helped make this possible and is giving hope to us and Paxton, and the other children around the world with CLCN6 mutations who are contacting us. We will share more updates with you as they become available. Please stay tuned and thank you all for your support, Paul, Kristin, Paxton, Aria Purdy
Paxton's Story: A Rare Genetic Battle Our son Paxton, recently diagnosed… Paul Purdy needs your support for Help Save Paxton's Life: A Rare Genetic Battle
