Israel Doctors Administer First WWOX Gene Therapy to 1 Child With WOREE Syndrome
Updated
Updated · MedicalResearch.com · Jun 9
Israel Doctors Administer First WWOX Gene Therapy to 1 Child With WOREE Syndrome
3 articles · Updated · MedicalResearch.com · Jun 9
Summary
An eight-month-old infant with WOREE syndrome received the first known WWOX gene replacement therapy under a compassionate-use pathway at Schneider Children’s Medical Center in Israel.
The treatment delivered a healthy WWOX gene directly into the brain through a cisterna magna injection using an AAV9 vector, aiming to restore gene function in neurons.
Early follow-up has been encouraging: the procedure was well tolerated, the child is clinically stable, and one month later severe seizures had not recurred.
Mouse studies had shown the approach could eliminate seizures and reverse neurological abnormalities, providing the proof of concept for moving into human use.
Mahzi Therapeutics is now preparing IND-enabling studies, but researchers said longer follow-up and more patients are needed to establish safety, durability and efficacy.