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 developed a method of using human induced pluripotent stem (hiPS) cells to generate three-dimensional neural floorplate organizers that are functionally active and capable of choreographing midline brain development.