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.
Researchers in the Wyss-Coray Lab are investigating a potential therapeutic antibody to treat lysosomal storage disorders and other related neurodegenerative diseases.
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.