Dose escalation pre-clinical trial of novel Dok7-AAV in mouse model of DOK7 congenital myasthenia
Judith Cossins, Imre Kozma, Claudia Canzonetta, Al Hawkins, David Beeson, Patricio V. Sepulveda, Yin Yao Dong
The paper evaluates a dose escalation pre-clinical trial of Amp-101, an AAV gene therapy, in a DOK7 CMS mouse model, showing improved neuromuscular junctions and muscle strength.
Brain Communications · Dec 24, 2024Peer-reviewedNeuroscience