NeurosciencebioRxivPreprint
AAV-mediated ARSA replacement for the treatment of Metachromatic Leukodystrophy
Shyam Ramachandran, Jeffery Ardinger, Jie Bu…·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.
2 citations