Technologies

Researchers at UC San Diego have developed a novel and highly efficient method for generating astrocytes from induced pluripotent stem cells (iPSCs). Using a well-characterized rare male Rett Syndrome (RTT) patient-derived and genome-edited isogenic lines, enriched populations of cortical neurons and astrocytes were generated. RTT derived astrocytes showed significant gene expression and physiological differences compared to control cells. Co-culture experiments revealed that RTT-derived astrocyte impaired neuronal dendritic arborization and spinogenesis in control cells. In contrast, control-derived astrocytes rescued morphological RTT neuronal phenotypes. Astrocytes used in these studies were characterized by immune-staining methods, followed by plating and neuronal enrichment.

Rett syndrome (RTT) is a devastating disease that affects 1 in every 10,000 children born in the United States, primarily females. RTT patients undergo apparently normal development until 6-18 months of age, followed by impaired motor function, stagnation and then regression of developmental skills, hypotonia, seizures and a spectrum of autistic behaviors. Rett syndrome is a rare disease that shares certain pathways with major developmental disorders such as autism and schizophrenia, increasing the potential impact. There is no cure for Rett syndrome and the animal model does not entirely recapitulate the human disease. Thus, having the possibility to screen drugs directly in human neurons is a major milestone.