Exploring therapeutic strategies for Infantile Neuronal Axonal Dystrophy (INAD/PARK14)
Guang Lin, Burak Tepe, Geoff McGrane, Regine C. Tipon, Gist F. Croft, Leena Panwala, Amanda Hope, Agnes JH Liang, Zhongyuan Zuo, Seul Kee Byeon, Lily Wang, Akhilesh Pandey, Hugo Jozef Bellen
The paper identifies ceramide accumulation, lysosomal expansion, and mitochondrial defects as root causes of INAD/PARK14 and tests therapeutic strategies including drugs and gene therapy.
eLife · Jan 16, 2023Peer-reviewedNeuroscience