Israeli Hospital Performs World's First Gene Therapy for Rare Genetic Epilepsy
An eight-month-old infant with a rare and devastating genetic epilepsy has become the first patient in the world to receive an experimental gene replacement therapy designed to restore the function of the WWOX gene directly in the brain. The landmark treatment was administered at Schneider Children's Medical Center of Israel in Petah Tikva, marking a major milestone in the development of precision genetic therapies for rare neurological disorders.
The condition, known as WOREE syndrome, is an ultra-rare disorder with only 60 to 90 genetically confirmed cases identified worldwide, and it is marked by drug-resistant seizures, profound developmental delay, and a sharply shortened lifespan. The breakthrough followed years of research led by Hebrew University of Jerusalem scientist Prof. Rami Aqeilan, who brought together scientists, clinicians, and biotechnology leaders from Israel and the United States. The infant's family chose not to release their names or any images.
Aqeilan described the achievement as the culmination of many years of research, explaining that an effort to understand the biological function of a single gene had grown into a potential therapeutic strategy for children facing one of the most severe forms of genetic epilepsy. He said the milestone demonstrated how fundamental research can advance from the laboratory toward new treatment options for patients with rare genetic diseases. The procedure represents the first clinical use of a gene replacement therapy aimed at restoring WWOX function in the brain of a child with the syndrome.
(JPost/VFI News)
Heavenly Father, we lift up this precious infant and family before You, asking that You would bless this treatment and grant healing, strength, and many years of life. We thank You for the gifts of wisdom and skill You have placed in Israel's doctors and researchers, and we ask that their work would bring relief to suffering children across the world. We pray that Israel would continue to be a light to the nations, a wellspring of healing and innovation that testifies to Your goodness. We ask Your comfort over every family walking through illness, and Your peace that surpasses all understanding, in Yeshua's Name.