World's First Gene Therapy for Rare Epilepsy Administered in Israel
Israeli hospital performs world's first experimental gene therapy for rare genetic epilepsy
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An eight-month-old infant in Petah Tikva, Israel, has received the world's first experimental gene therapy targeting the WWOX gene to treat a rare genetic epilepsy known as WOREE syndrome. This milestone, achieved at Schneider Children's Medical Center, offers hope for families affected by similar disorders.
- 01The infant diagnosed with WOREE syndrome began experiencing severe seizures at six weeks old due to a mutation in the WWOX gene.
- 02The gene therapy was developed through collaboration between Hebrew University of Jerusalem and Mahzi Therapeutics, utilizing an adeno-associated viral vector.
- 03Preclinical studies showed that the therapy restored WWOX expression and improved neurological outcomes in animal models.
- 04The therapy was administered directly into the infant's brain after regulatory approvals, and the child remained stable one month post-treatment.
- 05WOREE syndrome is characterized by severe developmental impairments and a high risk of premature death, with an average lifespan of two to four years.
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In a groundbreaking medical achievement, an eight-month-old infant has become the first patient worldwide to receive experimental gene replacement therapy for WOREE syndrome, a rare genetic epilepsy caused by mutations in the WWOX gene. Administered at Schneider Children's Medical Center in Petah Tikva, Israel, this innovative treatment aims to restore WWOX function directly in the brain. The infant, who experienced severe seizures starting at six weeks of age, was treated after extensive research led by Prof. Rami Aqeilan from Hebrew University of Jerusalem. The therapy uses an adeno-associated viral vector to deliver a healthy copy of the WWOX gene, showing promising results in preclinical studies. Following the treatment, the child was discharged from the hospital and remained clinically stable, with no recurrence of seizures reported. This milestone represents a significant step forward in developing personalized therapies for rare genetic neurological disorders, providing hope for families affected by similar conditions globally.
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This treatment represents a significant advancement in the field of genetic therapies for rare neurological disorders.
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