Israeli infant receives pioneering brain gene therapy for WOREE syndrome
TL;DR Summary
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.
- World's first WWOX gene therapy performed on infant in Israel The Jerusalem Post
- In first, Israeli doctors deliver gene therapy into brain of 8-month-old baby The Times of Israel
- Israeli doctors perform world's first gene injection therapy on infant i24NEWS
- Hebrew University: First WWOX Gene Replacement Therapy Administered to Child With Hereditary Seizures MedicalResearch.com
- Israeli infant receives first gene therapy for rare genetic epilepsy Azərtac
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