Stanford researchers have developed a genome engineering platform for generating genetically precise human cellular models of Lowe syndrome, a rare inherited disorder frequently associated with severe ocular complications, including early-onset glaucoma.
Researchers in the Wyss-Coray Lab are investigating a potential therapeutic antibody to treat lysosomal storage disorders and other related neurodegenerative diseases.
Stanford scientists have developed a prolonged drug-releasing formulation for delivering an iron chelation therapeutic to central nervous system in preterm births to prevent post-hemorrhagic hydrocephalus (PHH) in severe neonatal intraventricular hemorrhage (IVH)
Scientists in Sergiu Pasca's group at Stanford University have used patient-derived organoids, assembloids and in vivo transplantation to discover and validate an antisense oligonucleotide drug for the treatment of Timothy syndrome.
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.
Stanford inventors have developed a method to create spatially micropatterned vascularized structures that enable in vitro representation of human and animal biology in models such as cells, tissues, organs, and organoids.