Protei n anchoring-therapy indefectof extra cellar matrix at skeIeta muscle

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Congenital defects of ColQ cause endplate AChE deficiency and myasthenic syndrome. The intravenous administration of adeno-associated virus COLQto Colq-/-mice recovers motor functions, synaptic transmission, as well as the morphology of the NMJ. ColQ-tailed AChE is specifically anchored to NMJ. Furthermore, the in vivo injection of recombinant ColQ-tailed AChE protein complex into the gluteus maximus muscle of Colq-l- mice led to accumulation of AChE in non-injected forelimbs. We demonstrated for the first time in vivo that the ColQ protein contains a tissue-targeting signal that is sufficient for anchoring itself to the NMJ.