"The fact that injecting the human gene can rescue the zebrafish embryo is also an example of gene therapy that works really well, at least temporarily in the fish," adds Solnica-Krezel.
The zebrafish also makes it possible to pursue another, even more promising approach for developing a treatment for Menkes: Testing hundreds or thousands of compounds to see if any can restore proper copper-handling in Calamity mutants. If such a drug can be found, it would be a strong candidate for treating the disorder in human embryos.
"The zebrafish is the first animal model that allows us to watch the process of early vertebrate development and manipulate it. There is no reason why the same approach that we have used with copper cannot work for other nutrients as well," said Gitlin.
In fact, applying this approach to other nutrients and other types of birth defects is the goal of the Children's Discovery Institute, a major new initiative that Gitlin is heading as a joint venture between the St. Louis Children's Hospital and Washington University.