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health15h ago
Baby cured of rare epilepsy syndrome after doctors inject missing gene directly into his brain
- Eight-month-old patient with WOREE syndrome received brain-delivery gene replacement therapy.
- Therapy aimed to restore WWOX gene function to treat WOREE syndrome.
- The drug delivery was via a delicate neurosurgical procedure delivering a functioning gene copy into the brain.
- International collaboration and regulatory approvals supported the treatment.
- Preclinical data indicated gene replacement restored expression and improved outcomes in models.
- The child was discharged with no recurring seizures after treatment.
- Researchers emphasized this as a potential first-of-its-kind option for rare genetic diseases.
- WOREE is extremely rare, with only 60 to 90 cases identified worldwide.
- The gene therapy was licensed to a US biotech company, Mahzi Therapeutics.
- Experts hope this approach could lead to new treatments for similar genetic disorders.
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