Auburn University Research Provides Hope To Children Facing Deadly Genetic Disease

By Auburn University

Auburn University research and the heart-wrenching experience of an Opelika couple are providing hope to children facing the deadly genetic disease GM1 gangliosidosis.

The optimistic outlook is seen in an adorable 10-year-old girl named Jojo, who became the first patient to receive a gene therapy treatment, called AXO-AAV-GM1, during a human clinical trial this summer at the National Institutes of Health in Maryland. Auburn’s College of Veterinary Medicine and the University of Massachusetts Medical School developed the treatment that has moved from helping cats with GM1 to hopefully helping children.

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