US scientists have made a huge breakthrough in gene editing research, which they believe may help eradicate heart failure. For the first time, scientists were able to alter the DNA of defective embryos so they were no longer programmed to have heart failure.
They used gene editing tool Crispr-Cas9 to repair human embryos blighted by a single copy of a mutant gene, MYBPC3, that causes hypertrophic cardiomyopathy; a type of heart failure.
Scientists first fertilised donor eggs with sperm containing the defective gene through IVF. At the time of fertilisation, they applied the gene-editing tool that acts like a pair of scissors to remove the defective part of the gene.
Once the defective elements of the gene had been removed, the embryo replaced them with healthy elements.
Out of 58 embryos used, 42 (72.4 percent) were correctly fixed so that they no longer carried the heart failure mutation, which normally has a 50 percent chance of being passed on.
“Every generation on would carry this repair because we’ve removed the disease-causing gene variant from that family’s lineage,” said lead researcher, Dr Shoukhrat Mitalipov, from Oregon Health and Science University.
“By using this technique, it’s possible to reduce the burden of this inheritable disease on the family and, eventually, the human population."
Scientists believe this breakthrough could mean the end of heart failure; but the controversial research has raised ethical questions about human clinical trials. Some believe that such scientific advances could open the door for genetically modified and cloned babies.
The new research is particularly controversial because it involves modifying the nuclear DNA at the heart of the cell that also influences personal characteristics such as height, facial appearance, eye colour and intelligence.
The technique is still at the experimental stage, and there are no suggestions of human babies being created with the genetic modification for research purposes. This is illegal in both in the US and Britain, and even research on embryos is strictly limited.
In America, public funding for research that would involve destroying embryos is banned.
Congress has also banned the US Food and Drug Administration (FDA) from even considering the possibility of human clinical trials involving embryos with edited inherited genes.
However, lead researcher Dr Mitalipov has suggested that further research into gene editing could take place in the UK in the future, if laws were changed to allow it to take place.
