Israeli doctors have achieved a groundbreaking medical milestone by successfully delivering gene therapy directly into the brain of an eight-month-old infant suffering from a rare and life-threatening genetic form of epilepsy. The procedure, performed at Schneider Children’s Medical Center in Petah Tikva, marks the first time doctors have replaced a missing gene within a baby’s brain using a targeted gene therapy injection. The treatment focused on correcting a defect in the WWOX gene, a mutation associated with severe seizures, developmental delays, and a high risk of early death.
The pioneering therapy was built upon years of research conducted by Prof. Rami Aqeilan of the Hebrew University of Jerusalem. During the procedure, physicians delivered healthy copies of the missing gene directly into the child’s brain neurons through a single injection. According to reports, the infant has since been discharged from the hospital and remains in stable condition. Researchers hope the breakthrough could eventually provide new treatment options for children suffering from some of the most devastating inherited neurological disorders.
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