NeuroscienceMolecular TherapyPeer-reviewed
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…·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.
13 citations