
By using CRISPR, scientists were able to remove the gene behind hypertrophic cardiomyopathy.
Scientists were successful in using CRISPR, the gene editing technique, to remove a disease by correcting in a human embryo the gene responsible for causing hypertrophic cardiomyopathy. Although this is a remarkable breakthrough in genetical editing science, there are still many controversies on the matter.
Using CRISPR To Alter Genes, Still Very Much Banned
Using the CRISPR Cas-9 gene editing technology is a very risky action as the use of this method is still very much controversial. While some point out the ethical, moral, and religious impediments, others underline its still risky nature. Scientists are still uncertain as to what the long-term effects of using CRISPR might be, as the edited genes could start mutating in ways not even considered.
The United States, for example, has banned its use and the U.S. FDA is prohibited from conducting clinical trials involving inheritable genetic modifications.
The New Study and Hypertrophic Cardiomyopathy
This new study and breakthrough was carried out by an international team of scientists. Research involved taking the eggs of 12 healthy female donors and also sperm from a male donor carrying the MYBPC3 gene.
The latter is held responsible for the appearance of hypertrophic cardiomyopathy or HCM. This is a disease which affects the heart muscles and, in some cases, can lead to sudden cardiac death. Usually, HCM is a symptom-less disease until the moment of death.
Reportedly, it affects some 1 in every 500 people on a global scale. HCM is also held as being the most common cause of sudden death in young and healthy athletes.
The research team used CRISPR to remove the gene responsible for causing HCM before injecting the donor sperm into the eggs. Among the resulting cells, some 72 percent of them corrected themselves. They did so by using a non-mutated gene copy from the genetic material of the female donor.
Reportedly, there was no intention of intention of ever implanting these genetically modified embryos, so they were only left to grow for a few days before being destroyed. Still, the research team detected no adverse effects in the donor embryo’s unaltered DNA.
Study results were released in the journal Nature.
Image Source: Wikimedia


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