Thousands of diseases that are inherited, like Huntington's syndrome or cystic fibrosis, may not develop due to a new discovery in genetics.
The achievement, unveiled last week, is that scientists have been able to prove that it is possible to edit defective genes in the human embryo.
Using the CRISPR-Cas9 program, which resembles a find-and-replace command in a text editor, the scientists were able to alter the DNA in embryos so as to prevent future heart failure.
Fatal heart disease
The researchers focused on hypertrophic cardiomyopathy (HCM), a heart disease that affects one in five hundred people and can lead to sudden death. According to scientists, this is only one of 10 thousand known hereditary pathologies, which is caused by one gene. Potentially, many of them may not be developed by editing the corresponding genes in embryos.
"This study provides a new insight into the method that can be used in the treatment of thousands of hereditary genetic diseases affecting millions of people around the world," said the lead author of the method, Dr. Shukhrat Mitalipov from the Oregon University of Health and Science (USA).
“Each subsequent generation will be cured of the disease, because the gene that causes it will be removed from the family lineage. Using this technique, it is possible to reduce the burden of hereditary disease for a single family and, ultimately, for all of humanity,”added the scientist.
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Monogenic diseases
Monogenic diseases include hemophilia, Gaucher disease, and muscular dystrophy. As is the case with many other hereditary diseases, they can be detected during a DNA test.
The study, published in the journal Nature, also involved scientists from the Salka Institute (California) and Seoul National University. Scientists not involved in the study hailed the discovery as a turning point in genetic engineering.
“This appears to be a breakthrough in gene editing,” said Professor Daniel Bryson from the University of Manchester (UK).
"This remarkable study demonstrates how quickly the field of gene editing has changed since the CRISPR-cas9 system was chosen as the breakthrough 2015," added Professor Peter Browde of King's College London.
Three previous attempts to use the CRISPR system to create genetically modified human embryos were carried out in China, but with dubious results. This is the first study to provide safe and effective results.
Dmitry Utochkin