?-thalassemia is a devastating blood disorder caused by mutations in the HBB gene encoding ?-globin, where treatment involves lifelong, costly management of the resulting lack of hemoglobin and hemolytic anemia.
IPEX syndrome is a severe autoimmune disease with limited treatment options caused by mutations in the forkhead box protein 3 (FOXP3) gene, which plays a critical role in immune regulation.
Researchers at Stanford have developed gene editing methods for modifying hematopoietic stem and progenitor cells (HSPCs) to express truncated forms of the erythropoietin receptor (tEPOR).
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an efficacious therapy for patients with life-threatening leukemias, but its use has been hindered by the limited availability of donors with matching HLA. Graft manipulation by removing ??
Researchers at Stanford have developed methods to overcome the limited packaging capacity of adeno-associated virus (AAV) vectors and enable their use in integration of large transgenes.