An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health.
Mar 20 2024NIH/National Institute of Neurological Disorders and Stroke An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy , was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health . Currently, there is no treatment for GAN and the disease is usually fatal by 30 years of age.
Carsten G. Bonnemann, M.D., senior author and chief of the Neuromuscular and Neurogenetic Disorders of Childhood Section at the National Institute of Neurological Disorders and Stroke Mutations to the GAN gene result in an inability to break down intermediate filaments, which are cellular structures that make up the framework of nerve cell extensions called axons. Axons are essential for transmission of signals between brain cells. The disease name refers to the enlarged and bloated appearance of the axon under the microscope.
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