NCE (Year 4)

We have met almost all of the milestones and goals of this project. We have documented the efficacy of the Artemis vector in Artemis-deficient bone marrow stem cells by demonstrating that they are capable of maturing into T and B cells. We have also identified the optimal conditions for correcting the Artemis defect in these cells. Finally, we have demonstrated the safety of the vector and have shown that we can correct the defect in Artemis deficient mice.