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PIONEERING ACCESSIBLE GENE THERAPIES
FOR RARE DISEASES
UNDERSTANDING
GENE THERAPY
Gene therapy modifies or replaces faulty genes—the body’s instruction manual—to correct errors that cause disease. By delivering corrected genetic instructions to cells, it enables proper protein production and restores normal function.
Unlike traditional treatments that only manage symptoms, gene therapy targets the root cause, offering the potential for lasting and even curative effects.
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