8legs: Schneider children’s medical center of israel

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World's first WWOX gene therapy performed on infant in Israel | The Jerusalem Post

An eight‑month‑old with WOREE syndrome received an experimental WWOX gene therapy directly in the brain at Schneider Children’s Medical Center.
The treatment was delivered at Schneider Children’s Medical Center of Israel in Petah Tikva as part of a compassionate‑use program.
Researchers used an AAV9 vector to deliver a healthy copy of the WWOX gene to neurons, with preclinical studies supporting the concept.
Mahzi Therapeutics is advancing the program toward clinical application and coordinating translational and regulatory activities.
WWOX‑related epileptic encephalopathy causes early‑onset, drug‑resistant seizures and severe developmental impairment.
Early follow-up showed no recurrence of severe seizures during the initial observation period after treatment.
The program builds on HUJI research linking WWOX to nervous system development and function.
The initial success is described as a milestone toward personalized therapies for rare genetic epilepsies.
The infant's condition originated from a genetic defect common in Yemeni Jewish ancestry but with variants worldwide.
The therapy represents a collaboration among Israeli and US scientists and regulatory partners.

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