?-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.
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 in Prof. Michael Lin's laboratory have developed a viral-based cancer therapy platform that could be used for targeting treatment to cancer cells with aberrant signaling in EGFR or HER2 pathways.
Researchers in Prof. Stephen Quake's laboratory have developed a CRISPER-Cas-based targeted endonuclease system designed to treat latent viral infections by attacking the viral genome.
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.