Zebrafish in the clinic

Precision medicine has ushered in a new era when it comes to the treatment of certain cancers. But the potential for transformation extends into far more diseases.

In a case study published yesterday in Nature Medicine, researchers at the Children’s Hospital of Philadelphia detailed how they used zebrafish to save the life of a boy with a rare, complex lymphatic disorder. In short, they identified the mutation causing the boy’s illness; engineered the mutation into zebrafish; waited for the fish to develop a piscine version of the boy’s disease; and then tested multiple drugs on the fish — until they found one that worked.

“I think this approach definitely could be used clinically,” one medical geneticist told STAT’s Sharon Begley.

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