Executive Summary Gene therapy represents one of the most promising advances in the treatment of genetic epilepsies. Technologies including adeno-associated virus (AAV) vectors, antisense oligonucleotides (ASOs), gene replacement, gene editing, and gene regulation have the potential to transform the lives of patients with developmental and epileptic encephalopathies (DEE). Yet an important scientific and commercial question remains: Will correcting the genetic mutation be sufficient to reverse epilepsy once abnormal brain networks…
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