Israeli doctors perform world's first gene injection brain therapy on infant

Doctors at Clalit-Schneider Children's Medical Center in Israel have performed a world first, injecting a healthy gene directly into the brain of an eight-month-old baby born with a fatal genetic neurological disorder. The procedure had never previously been attempted on a human being.

The breakthrough united an unlikely team: an Israeli physician who recognized the rare condition, an Arab scientist from the Hebrew University of Jerusalem who originally developed the therapy, and the Israeli-born CEO of the American biotech company that brought it to clinical use. Together they navigated emergency regulatory approvals across multiple countries to give the infant from an ultra-Orthodox Jewish family a chance at survival.

The case began when Dr. Naama Ornstein, head of the genetics unit at Clalit-Schneider, recognized the rare disorder after the baby was admitted with seizures and severe developmental decline. Years earlier she had treated another child with the same condition, but no treatment was available at the time, and that child died. Determined not to lose another patient, she reached out to Professor Rami Aqeilan, the Arab researcher at Hebrew University whose academic discovery had since been developed into a clinical therapy.

In a delicate neurosurgical procedure, Dr. Ido Ben Zvi injected a functioning copy of the missing gene directly into the baby's brain using a specially engineered viral vector. Dosage calculations were among the most sensitive aspects of the procedure, requiring precise tailoring to the infant's brain size. The operation required special emergency approvals from Israeli health authorities and international oversight bodies.

The baby has since been discharged and is being closely monitored at home. "This was much more than a medical procedure," said Dr. Ornstein. "It was a fight for a child's chance to smile, develop, and live. Families often ask us, If a child is missing a working gene, why can't we simply give them one? Usually, we have no answer. This time, for the first time in the world, we could."

The case is already being described at Clalit-Schneider as a landmark moment in the future of personalized genetic medicine and a rare story of science, humanity, and cross-border cooperation overcoming every barrier to save a child's life.