Uncovering targets and molecular pathways for personalizing treatment in epilepsy

Therapy in the near future will likely be based predominantly on specific small molecules or gene therapies, such as antisense oligonucleotides. Among currently used allele-specific therapeutic designs, only some loss- or gain-of-function pathological variants are expected to profit from them. Allele-specific gene therapy may be a promising treatment for those diseases that would not benefit from currently used allele-nonspecific designs.