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The first US trial for using CRISPR [KRIS-per] to erase a heritable heart condition from human genes has succeeded.
In recent years, CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, has been gaining popularity as a gene-editing tool, or a way to make specific changes directly on an organism’s genes. In fact, it has been used to delete and add genetic information in mice.
It is only recently that CRISPR has been used in human clinical trials. China pioneered human trials of CRISPR in 2016 when it was used to disable a lung cancer gene from a patient. Six months after, another team of Chinese researchers conducted CRISPR trials to remove different cancer genes from 20 patients.
The first US CRISPR trial involved creating embryos that carry a gene for hypertrophic cardiomyopathy (HCM), a heritable condition that causes swelling of the heart muscles. In the experiment, doctors used CRISPR to cut the HCM gene from the embryos. The researchers were astonished by the success rate of the experiment, as HCM was removed in 72% of the embryos.
Though many have a positive outlook on CRISPR, several countries still have legal restrictions on gene-editing. A 2014 research showed that laws in most countries, including the United States, ban gene-editing on humans. Meanwhile, others allow trials only if they follow certain guidelines.
Vigilance against gene-editing arises partly from the fear that it will lead to the creation of “designer babies.” Some critics are concerned that someday, gene-editing would not only be used to eliminate genes for diseases but also to add desirable traits like high intelligence or athletic abilities to babies. However, some experts claim that there is no need to worry because the technology and knowledge required to do that is far too advanced.