Scientists at the Sun Yat-sen University in China have managed to conduct DNA surgery on an embryo to remove a genetic disease from it.
Beta-thalassemia is a dangerous condition that can cause significant issues in infants including anemia, skeletal abnormalities, and even death. A single point mutation causes it. That means only one base pair out of over three billion base pairs in the whole DNA strand is responsible for beta-thalassemia. This made it an excellent candidate for the revolutionary new procedure.
Base Editor and its Contribution in Curing the embryo
The team used an adaptation of a CRISPR DNA snipping tool which they call a base editor. Using this amazing tool, they edited the single mutated base within the DNA of that embryo. This fixed the faulty gene and would have prevented the disease if the embryo had been allowed to develop.
Feats of DNA surgery like this were thought to be completely impossible until very recently.While some genetic disorders like breast cancer can involve a confluence of many genes, there are over six hundred diseases that only include a single one. In theory, a dangerous disease like cystic fibrosis or muscular dystrophy could be cured the same way this study cured beta-thalassemia.
While nobody can argue the benefits of fixing genetic disorders, some fear the ethical implications involved. Especially those regarding “designer babies” and their social consequences. Others fear the possible unintended consequences of gene modifications and their longer-term effects. Genes are complicated, and many genes influence many genetic diseases. In fixing one disease, scientists could cause a multitude of other maladies.
Despite the controversy, Chinese researchers have been investigating genetic modification for years, and in 2017 the US decided to allow limited research into genetic editing, as well. While it is possible that changing the human genome might have its downsides, nobody can argue that its benefits could overshadow anything the medical community has come up with to this point.