Israeli infant receives pioneering brain gene therapy for WOREE syndrome
An eight‑month‑old in Israel became the world’s first patient to receive an experimental brain gene‑replacement therapy aimed at restoring WWOX function to treat WOREE syndrome, delivered directly into the brain via an AAV9 vector at Schneider Children’s Medical Center. The procedure followed years of research led by Prof. Rami Aqeilan and supported by Mahzi Therapeutics; one month after treatment the baby was stable, had no seizures, and was discharged, but long‑term safety and efficacy require ongoing follow‑up.