A research done on a Zebrafish showed that a genetic defect may be prevented by injecting a customized genetic patch on embryos. The genetic mutation is corrected at the early stage, enabling the embryos to develop normally. This research can lead to a similar study on humans. Zebrafish are vertebrates, and develop in a similar way like humans, and its transparent appearance enables researchers to observe the development of embryos. The genetic defect that was observed and prevented was the zebrafish model of Menkes disease. Children with this disease have seizures, abnormal bone development and kinky, colorless hair, among others, and they die before they reach 10 years of age. The researchers, headed by Erik C. Madsen, first author and an M.D./Ph.D. student in the Medical Scientist Training Program at Washington University School of Medicine, developed morpholinos that prevent the gene mutation.
Consider the genetic code as a book, and someone has put in random letters or gibberish in the middle of the book,” Madsen said. “To be able to read the book, you have to ignore the gibberish. If we can make cells ignore the gibberish, or the splicing defect, the fetus can develop normally.
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