Stanford researchers transplant human brain organoids into engineered mice
Scientists at Stanford have implanted lab-grown human cortical organoids into genetically modified mice lacking most of their cortex, creating a model to study neurodevelopmental disorders.
Researchers at Stanford University have successfully grafted laboratory-grown human cortical organoids into bioengineered mice that were bred to lack most of their cerebral cortex, according to a study published in Nature. The organoids, derived from human skin cells reprogrammed into stem cells, formed three-dimensional clusters that mimic specific brain regions and were transplanted into the mice, where they survived, grew and formed functional connections with the host nervous system.
"In these mice, the human grafts generated a broad diversity of cortical cell types and established functional connections throughout the mouse nervous system," said senior author Professor Sergiu Pasca. He added that the animals retain a mouse nervous system but contain a larger volume of human cortical tissue that integrates and forms connections, providing a platform to study human neural cell types and developmental processes.
The model is intended to facilitate research into conditions such as autism, schizophrenia, cerebral palsy and epilepsy by allowing scientists to observe how disease-associated genetic changes affect neural development and to test potential interventions. Alison Singer, president of the Autism Science Foundation, described the approach as a step toward precision medicine that could enable study of individual genetic profiles in brain tissue.
Pasca noted that the technique offers a way to investigate neurodevelopmental and prenatal disorders at the molecular and cellular level, which has been challenging due to the complexity of the human brain and ethical constraints on studying living human brain tissue.