Abstract
SnRNA systems targeting SCN1A or SYNGAP1 RNA sequences are disclosed herein. Further disclosed are methods of treating genetic epilepsies such as Dravet Syndrome or SYNGAP1-related epilepsy.
Full Text
What is claimed is:
SnRNA systems targeting SCN1A or SYNGAP1 RNA sequences are disclosed herein. Further disclosed are methods of treating genetic epilepsies such as Dravet Syndrome or SYNGAP1-related epilepsy.
Timeline
Filed
04/22/2026Published
08/13/2026Granted
Not AvailableIPC Codes(2)
C12N 15/113:Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/86:Viral vectors