A mechanistic classification of GNAO1 mutations links Gβ γ sequestration to hyperkinetic disease and treatment response
Gabrielle Soden, Amy Robichaux‐Viehoever, Kaitlin Soden, Jeff Laux, Henrik Gunnar Dohlman, Daniel J. Christoffel
This study classifies GNAO1 mutations into functional categories and evaluates therapeutic strategies for NEDIM and DEE, finding that specific mutations are linked to hyperkinetic symptoms and that small molecules reduce hyperlocomotion.
bioRxiv · Oct 29, 2026 · PreprintPharmacology & Toxicology