Neuronal genetic rescue normalizes brain network dynamics in a lysosomal storage disorder despite persistent storage accumulation
Rebecca C. Ahrens‐Nicklas, Luis Tecedor, Arron F Hall, Owen Kane, Richard J. Chung, Елена Лысенко, Eric D. Marsh, Colleen S. Stein, Beverly L. Davidson
This study shows that restoring Cln3 in neurons normalizes brain network dysfunction in CLN3 disease despite persistent storage accumulation, suggesting neuronal loss-of-function, not storage, drives neurologic symptoms.
Molecular Therapy · Apr 5, 2022Peer-reviewedNeuroscience