?-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.
Stanford researchers in the Goldberg lab have developed a novel method for targeted gene therapy delivery to retinal astrocytes for the treatment of glaucoma and other optic neuropathies.
Stanford researchers in the Goldberg lab have discovered two novel gene therapy targets for the treatment of glaucoma and other optic neuropathies. Glaucoma is the leading cause of irreversible blindness world-wide, affecting millions of adults in the United States alone.
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.
Researchers in Prof. Mark Kay's laboratory have developed recombinant adeno-associated viral (AAV) capsid proteins that transduce human primary hepatocytes at high efficiency in vitro and in vivo.