Gene editing technique successfully corrects mutation in human embryos

Researchers in the United States have successfully used a gene editing technique in early stage human embryos to correct a mutation that causes hypertrophic cardiomyopathy, a report in Nature has confirmed.1

The embryos in the study were not allowed to develop for more than a few days and were never intended to be implanted into a womb. Further research is needed to establish the safety of the method before it can be adopted clinically. However, if the technique proves to be safe, it could potentially be used for other genetic disorders such as cystic fibrosis or Huntington’s disease.

Hypertrophic cardiomyopathy is a common inherited condition affecting one in 500 people, and it is the most common cause of sudden death in otherwise healthy young athletes. In 40% of families with hypertrophic cardiomyopathy it is caused by a single copy of a mutant MYBPC3 gene.

The research team produced zygotes by fertilising healthy …