ube-cel TMX

A therapeutic approach where an individual’s own bone marrow stem cells are removed from their body, modified ex vivo, or outside the body, and returned to the body with a replaced copy of the missing or nonfunctional gene (UBE3A in this case). Once the bone marrow cells are transplanted back into the individual, they go to the bone marrow to grow and repopulate. The goal is for those cells to continuously supply the body with a healthy version of the gene. These cells can cross from the blood to the brain, through the blood brain barrier (BBB). Once they cross the BBB they become a cell type called microglia and secrete the UBE3A protein throughout the brain for neurons to take it up and use it.