Dose escalation pre-clinical trial of novel DOK7-AAV in mouse model of DOK7 congenital myasthenia by Cossins, J, Kozma, I, Canzonetta, C, Hawkins, A, Beeson, D, Sepulveda, P, Dong, YY

“…EMG showed that the treated model mice had decrement of compound muscle action potential on repetitive nerve stimulation, which indicates defective signalling at the neuromuscular junction. However, male models treated with 1 × 1014 vg/kg showed the least decrement that was not statistically different from wild-type littermates. …”