Stem cells are generally influenced by a microenvironmental niche, typically comprised of epithelial and mesenchymal cells and extracellular substrates. Many attempts have been made to produce culture systems that mimic normal intestinal epithelial growth and differentiation.
?-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.
Researchers at Stanford University have developed a method which integrates cell barcoding and high-throughput sequencing to quantify tumor growth in genetically engineered mouse models of human cancer (called 'Tuba-seq” for Tumor barcoding coupled with seq
Researchers in Prof. Mark Kay's laboratory have developed variant AAV (adeno-associated virus) vectors with specificity and high transduction efficiency for pancreatic alpha- and beta- islet cells.
The Hu Lab at Stanford has developed a neuroprotective gene therapy for treating glaucoma and other optic neuropathies. Their gene therapy AAV vector expresses NMNAT2 operably linked to a retinal ganglion cell-specific promoter (mSngc).
Stanford scientists have developed a neuroprotective, adeno-associated virus (AAV) gene therapy vector that expresses a mutant form of HDAC4 or a fragment of HDAC4 with novel applications to retinal and neurologic diseases, including glaucoma and other retinal ganglion cell di
Stanford inventors have engineered an adeno-associated virus (AAV) variant on the existing LK03 platform that enables this highly efficient primate-specific serotype for use in rodent preclinical studies.
Adeno-associated virus (AAV) vectored products are currently leading candidates for gene therapy applications with multiple approved products and many more in clinical trials.
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).
Engineered viruses have great potential as cancer treatments. However, the only currently approved viral therapy, T-vec (Talimogene laherparepvec), suffers from off-target effects that limit its use to intratumoral injection.
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.