N-of-1 AAV gene therapy stops WWOX epilepsy seizures in an infant

An eight-month-old with WWOX epilepsy became seizure-free after an intrathecal AAV delivered a working gene copy, extending n-of-1 medicine beyond ASOs

Amazing things in gene therapy are happening every day. FIH intrathecal AAV for WWOX epilepsy - working copy of the gene into the cisterna magna of an 8mo at Schneider, baby is one month seizure free. Effectively a milasen-style n-of-1 but