Targeting a disease is not so easy sometimes, even though medicine is so advanced in the past couple of decades. However, medicine has achieved targeting diseases and disorders even before the babies are born, when they are still fetuses. The implications that come after targeting these kinds of issues are big, but it seemed that solving them was quite impossible. Luckily for humanity, a team of researchers have achieved what seemed like the impossible. A collaboration between American and South Korean institutions brought a success in research of a faulty DNA embryo. Researchers managed to remove the gene malfunction that causes a deadly heart disease. This disease can pass to the family members easily, but that will not happen again. Science needs to celebrate!
With this technology, scientists could stop diseases like this from ever occurring in the families.
This is how the research team worked.
The team focused on hypertrophic cardiomyopathy, which is a common heart disorder. This disorder affects one in 500 people, and can even cause a sudden heart failure. It all comes down to an error in a single gene. This can pass on to the family by a 50/50 chance. When scientists”corrected” the error, they can prevent the disease from ever occurring.
Researchers carried out the experiment at Oregon Health and Science University and the Salk Institute, along with the Institute for Basic Science in South Korea. The research team allowed for the embryos to develop for 5 days before stopping the experiment.
This is what the researchers did. They injected sperm of a man with hypertrophic cardiomyopathy into healthy donated eggs. Then, they used the Crispr technology to correct it during development. Every case was not successful, however. Also, the safety of the procedure still needs to be verified, but after their work a stunning 72% of embryos were free from mutations.
Speaking to the BBC, Dr. Shoukhrat Mitalipov said:
“Every generation on would carry this repair because we’ve removed the disease-causing gene variant from that family’s lineage. By using this technique, it’s possible to reduce the burden of this heritable disease on the family and eventually the human population.”
After multiple attempts across the world, this is the very first successful one that has no side-effects. Apparently, there are about 10,000 genetic disorders by a single mutation. Scientists could repair all of them with the same technology.
“A method of being able to avoid having affected children passing on the affected gene could be very important”, Professor Robin from the Francis Crick Institute, explained. “In terms of when, definitely not yet. It’s going to be quite a while before we know that it’s going to be safe.”
The success of this research is not the only thing that is debatable. There are many questions arising, that are not only scientific. Darren Griffin, a professor of genetics, said: “Perhaps the biggest question is whether we should be physically altering the genes of an IVF embryo at all. Also, there are moral questions arising, as well as the fine-tuning of the procedure. However, it is a great success in scientific terms.