Alabama NewsCenter — Auburn University research providing hope for children with deadly disease GM1

By Charles Martin
Auburn University

Jojo is experiencing life like most 13-year-old girls: staying up later, watching movies, shopping and baking cookies. Now she has more liberty, but it’s a different kind of freedom.

She uses a walker, but she and her family see it as freedom, a remarkable accomplishment compared to her six months of not being able to walk prior to receiving a gene therapy treatment for her disease.

Jojo has GM1 gangliosidosis, an inherited disorder that progressively destroys nerve cells in the brain and spinal cord and is estimated to occur in about one in 100,000-200,000 newborns. But her mother sees steady improvement in Jojo, who was the first child in a clinical trial that has its origin in Auburn University research.

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