Creating mini-brains from stem cells reveals a new, promising treatment for a devastating childhood disease

Article excerpt

Dutch and American researchers used miniature brain organoids grown from patients' own stem cells to unlock a potential treatment for a rare, devastating childhood neurodegenerative disease linked to mutations in the DHDDS gene. The condition, characterized by tremors, seizures, and progressive loss of coordination and learning ability, typically emerges in early childhood and has offered no slowing options until now. By modeling the disease in these lab-grown tissues, scientists identified both how the genetic defect wreaks havoc on developing neurons and a promising therapeutic avenue. The findings offer hope to families who have long heard that nothing could be done.