| abstract |
Alternative splicing events in the SCN1A gene can lead to unproductive mRNA transcripts which, in turn, can lead to aberrant protein expression. The object of the present invention is to provide therapeutic agents capable of targeting alternative splicing events in the SCN1A gene and modulating the expression level of functional proteins in patients with Dravet's syndrome and / or To inhibit aberrant expression of proteins, these therapeutic agents can be used to treat a disease state caused by SCN1A, SCN8A or SCN5A protein deficiency. |