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

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...

Bibliografiset tiedot
Päätekijät:	Cossins, J, Kozma, I, Canzonetta, C, Hawkins, A, Beeson, D, Sepulveda, P, Dong, YY
Aineistotyyppi:	Journal article
Kieli:	English
Julkaistu:	Oxford University Press 2025

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