First-in-the-world gene therapy delivers missing gene directly to infant’s brain, marking historic milestone in precision medicine
Teona Gherasim
Jun 11 2026 An eight-month-old baby from Israel has become the first human ever to receive an experimental gene therapy designed to replace a missing gene responsible for a devastating neurological disease that until now offered no hope of survival. From right to left: Dr. Dror Kraus, Senior Physician, Neurology Unit and Epilepsy Specialist, Clalit-Schneider Children’s Medical Center; Dr. Naama Ornstein, Head of the Genetics Unit, Clalit-Schneider Children’s Medical Center; Prof Rami I. Aqeilan.
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