Altered lysosomal biology impairs motor neuron survival via TFEB dysregulation in spinal muscular atrophy
Ines Rosignol, Zeynep Dokuzluoglu, Antonio Caldarelli, Ana‐Maria Oprişoreanu, Sofiia Ushakova, Tohid Siddiqui, Rahul Grover, Hannes Guerlich, Björn H. Falkenburger, Stefan Diez, Tobias Graß, Catherina Gwynne Becker, Natalia Rodríguez‐Muela
The paper shows that TFEB activation restores lysosomal function and improves motor neuron survival in SMA, highlighting its potential as a therapeutic target.
bioRxiv · Jul 7, 2025 · PreprintNeuroscience