AAV-mediated ARSA replacement for the treatment of Metachromatic Leukodystrophy
Shyam Ramachandran, Jeffery Ardinger, Jie Bu, MiAngela Ramos, Lilu Guo, Dhiman Ghosh, Mahmud Hossain, Shih-Ching Chou, Yao Chen, Erik Wischhof, Swathi Ayloo, Roger Trullo, Yuxia Luo, Jessica M. Hogestyn, Daniel DuBreuil, , Christian Mueller
This study develops an AAV vector for ARSA gene replacement in MLD, showing improved brain biodistribution and therapeutic effects in mice and non-human primates.
bioRxiv · Mar 12, 2025 · PreprintNeuroscience