INTERFERENCE RNA FOR REDUCING THE HUMAN NAV1.6 CHANNEL EXPRESSION.

摘要

Experimental evidence is shown for the first time about the effectiveness of a directed shRNA against the human Scn8A gene expression (NaV1.6), capable of decreasing the mRNA abundance, and the amount of total protein of the NaV1 channel 1.6 corresponding to the gene transcription and translation, respectively; as well as of the current density generated by the functional activity of the NaV1.6 channels present in the plasmatic membrane. Due to the lack of pharmacological tools to reduce the activity of this channel, the present invention represents an alternative to fulfill such purpose under experimental conditions in the investigation and potentially as a therapeutic strategy in the CaCu treatment and other pathologies characterized by the deregulated expression of the NaV1.6 channel.