First human gene embryo editing could stop genetic diseases

Posted July 28, 2017

The effort, led by Shoukhrat Mitalipov of Oregon Health and Science University, involved changing the DNA of a large number of one-cell embryos with the gene-editing technique CRISPR, according to people familiar with the scientific results. "So far as I know this will be the first study reported in the USA", says Jun Wu, a collaborator at the Salk Institute, in La Jolla, California, who played a role in the project.

In December 2015, scientists and ethicists at an worldwide meeting held at the National Academy of Sciences (NAS) in Washington said it would be "irresponsible" to use gene editing technology in human embryos for therapeutic purposes, such as to correct genetic diseases, until safety and efficacy issues are resolved.

The embryos, which were modified to test the feasibility of fixing known disease-causing genes, were terminated days after the experiment. Although the report said that "many tens" of embryos were created in the experiment, they were never meant to be implanted into a womb and were only allowed to develop for a few days. This gene modification process is referred to as germline engineering, since a person carrying the altered genes would pass the changes on to any offspring they had, via their own eggs or sperm (germ cells).

Mitalipov's group appears to have overcome earlier difficulties by "getting in early" and injecting CRISPR into the eggs at the same time they were fertilized with sperm.

Don't expect a new generation of gene-edited people in the USA, though: Any local efforts to turn edited IVF embryos into babies have, so far, been blocked by Congress. They significantly reduced mosaicism.

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"It is proof of principle that it can work".

It involves technology called "CRISPR" to alter genes in embryos. The scientists had no plans to actually allow them to develop, but the work is a significant step forward for those who support continued development of CRISPR and the eventual implementation of the technique to correct flaws in embryos carried to full term.

Federal regulations have barred scientists in the United States from any efforts to turn an editing embryo into a baby.

Many are concerned the technology could be used to make so-called "designer babies". In a report this year, National Academy of Sciences (NAS) and the National Academy of Medicine said scientific advances made gene editing in human reproductive cells "a realistic possibility that deserves serious consideration".