Genes for hereditary heart disease removed from human embryo

US researchers have removed a hereditary heart disease from a human embryo thanks to a CRISPR method. This is the first time a human embryo has been edited within the United States. Study paves way for new era in medicine.

The first-ever experiments using CRISPR-Cas9 technology on a human were conducted in China. A patient suffering from an aggressive form of lung cancer was given altered com immune cells with the hope that modifying one of the genes deal with cancer. Also in China, this method was first used to modify human embryos.

No one else has conducted similar experiments in not due to in technological, but moral and ethical. It seems that scientists from the Middle Kingdom have no major problems with this. And it has made China the world leader in research using CRISPR-Cas9 in humans. Only now to study the editing of the gene in human beings was joined by scientists from the US.

The gene editing technique CRISPR-Cas9 has been hailed by researchers as a breakthrough, as one of the most powerful tools of co modern medicine. It has removed the HIV virus from the human body, altered the external appearance of wasps and even written into the DNA of a living bacteria film. However, its use comes with considerable risks. It was reported at the end of May that the method could lead to dangerous mutations in the genome.

US researchers from Oregon Health joined the study of this method & Science University (OHSU) in Portland. They have successfully edited the DNA of human embryos in order to remove an inherited heart disease from them – hypertrophic cardiomyopathy. The disease involves spontaneous left ventricular hypertrophy or segmental thickening of the walls of the heart chamber. The disease does not give a symptom and in some cases is associated with sudden death. Although diagnosed can be treated.

According to the researchers, if one of the parents has a defective copy of a gene called MYBPC3, then there is a 50 percent chance that the genetic mutation will be passed on to the child. It was this gene that was targeted in the OHSU scientists’ study. This is at least the second pr ba modifying an embryo in human in the world using CRISPR, but this study is the first successful pr b by removing the disease in vivo (from the Latin. on a living, inside a living organism).

Scientists succeeded in removing the MYBPC3 gene in 42 of 58 embryos which re used for the study. This gives 72 percent. effectiveness. But the researchers say the method can still be improved, and they hope to achieve 100 percent success rate in the long run. The embryos were removed after a few days.

Researchers also need to make sure that the gene modifications do not cause unintended mutations in other parts of the genome. Of the 42 embryos, with which rych the defective gene was removed, only one showed signs of an unplanned mutation.

The study raises ethical controversy, and debates on the subject will no doubt continue. The solution to the problem is to edit the gene them of the embryo, and the com ova and sperm. This approach is supposed to be more morally justified according to the scientists. But despite the controversy, the experiment shows that it is possible to correct defects in the human body at an early stage of development.