Dose escalation pre-clinical trial of novel DOK7-AAV in mouse model of DOK7 congenital myasthenia

Congenital myasthenic syndromes are a group of inherited disorders characterized by defective neuromuscular transmission and fatigable muscle weakness. Causative mutations have been identified in over 30 genes, including DOK7, a gene encoding a post-synaptic protein crucial in the formation and stab...

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Main Authors: Cossins, J, Kozma, I, Canzonetta, C, Hawkins, A, Beeson, D, Sepulveda, P, Dong, YY
פורמט: Journal article
שפה:English
יצא לאור: Oxford University Press 2025