Creating Mini-Brains From Stem Cells Reveals a New, Promising Treatment for a Devastating Childhood Disease

Researchers map rare DHDDS disease mechanism using lab-grown mini brains

Variants in the DHDDS gene cause a severe neurodegenerative condition, characterized by tremors, seizures, coordination and learning difficulties, usually manifesting in early childhood. This Parkinson's-like condition is extremely rare, and until recently, parents were told that there was nothing that could be done to slow down its progression.

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

Variants in the DHDDS gene cause a severe neurodegenerative condition, characterized by tremors, seizures, coordination and learning difficulties, usually manifesting in early childhood. This Parkinson's-like condition is extremely rare, and until recently, parents were told there was nothing that could be done to slow its progression. But now, researchers from the Netherlands and the U.S. who created "mini brain" models from patients' own cells to test new therapies have found not only the mechanism of the disease, but also that a naturally occurring form of vitamin B3 (nicotinamide mononucleotide, or NMN) holds significant promise in slowing disease progression.

Mini-Brains Grown from Stem Cells Offer Hope for Treating Devastating

A Breakthrough in Combating DHDDS-Related Neurodegenerative Disease: How Mini-Brains Illuminate Pathways to a Vitamin B3 Therapy In a groundbreaking advancement within neurogenetics, researchers have uncovered promising therapeutic avenues for a devastating congenital condition triggered by mutations in the DHDDS gene. This rare disorder, manifesting early in childhood, leads to progressive neurodegeneration characterized by movement tremors, se…