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And she even described an extremely rare disease: type IV glycogen storage disease AKA Andersen’s disease.

In April 2018, Ultragenyx’s gene therapy to treat Glycogen Storage Disease Type 1a got the all clear.

The p53 problem, however, might affect other products that companies hope to develop via gene correction, including glycogen storage disease, cystic fibrosis, and severe combined immunodeficiency.

Only 1 year old, she already has been tasked with looking out for LeBeau, who was diagnosed with glycogen storage disease type Ia when he was 10 months old.

Other diseases in which correcting a small percentage of cells might make a big difference include glycogen storage disease and ornithine transcarbamylase deficiency, an inherited disorder that causes ammonia to accumulate in the blood.