First-in-the-World Gene Therapy Delivers Missing Gene Directly to Infant's Brain, Marking Historic Milestone in Precision Medicine
Researchers at the Hebrew University of Jerusalem successfully delivered a missing gene directly into the brain of an infant using gene therapy, marking the first such procedure in the world. The treatment targeted WWOX-related epileptic encephalopathy, a rare and severe neurological disorder, and demonstrated dose-dependent, durable rescue effects in prior animal models.
Scientists at the Hebrew University of Jerusalem’s Lautenberg Center for Immunology and Cancer Research have achieved a historic milestone in precision medicine by performing the world’s first gene therapy that delivers a missing gene directly into an infant’s brain. Led by Prof. Rami Aqeilan, the team targeted WWOX-related epileptic encephalopathy, a devastating neurological condition characterized by severe seizures and developmental delays. The therapy involved introducing the WWOX gene, which is critical for brain development, directly into neurons of an affected infant. Previous research by the team, published in journals like *EMBO Molecular Medicine* and *bioRxiv*, confirmed the approach’s effectiveness in animal models, showing dose-dependent and lasting improvements in symptoms. These studies also linked WWOX deletions to epilepsy and myelin defects, reinforcing the gene’s role in neurological health. The breakthrough builds on earlier work, including a 2021 study in *Brain* that demonstrated how neuronal deletion of WWOX in mice replicated human epilepsy and myelin disorders. The latest clinical application follows rigorous preclinical testing, including a 2026 *Neurobiology of Disease* paper outlining WWOX’s molecular mechanisms and therapeutic potential. While details about the infant’s identity remain confidential, the procedure represents a paradigm shift for treating genetic neurological disorders. The Hebrew University’s spokesperson, Danae Marx, emphasized the collaboration between basic science and clinical innovation, highlighting the potential to translate lab discoveries into life-saving treatments. Further studies will assess long-term outcomes and scalability for other genetic conditions. This achievement underscores the growing role of gene therapy in addressing previously untreatable diseases, particularly those with neurological origins. The research was supported by peer-reviewed publications, including preprints on *bioRxiv* and studies in *PubMed*-indexed journals, ensuring transparency and scientific rigor.
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