CRISPR corrects mutation in human embryos

CRISPR corrects mutation in human embryos

"Superior" designer babies born into the upper class with genetically modified physical appearances and intelligence could be just years away, a British geneticist has warned, after scientists announced they had used gene editing to fix a mutation in human embryos.

OHSU said the study's research adhered to guidelines established by the university's Institutional Review Board and additional ad-hoc committees set up for scientific and ethical review.

The process is still only laboratory research.

Dr David King, director of the Human Genetics Alert, which opposes all alterations of the human genome, said: "If irresponsible scientists are not stopped, the world may soon be presented with a fait accompli of the first GM baby".

CBS News chief medical correspondent Dr. Jon LaPook said the technology raises "huge ethical concerns".

The team created a gene-editing tool, named CRISPR-Cas9, that acts like a pair of molecular scissors to find that mutation. He wasn't involved in the new research and praised it as "quite remarkable". As a result, only 28% of the resulting embryos carried genes for hypertrophic cardiomyopathy.

Thousands of inherited diseases such as Huntington's Disease and Cystic Fibrosis could eventually be nipped in the bud after a major breakthrough in gene editing. They explicitly urged caution when it came to "germ-line" editing, since the heritable changes should be considered for compelling reasons.

The scientists said that CRISPR-Cas9, combined with in vitro fertilization, could be helpful for many other genetic diseases once more clinical studies are carried out.

The new report was published August 2 in the journal Nature. The scientists targeted a mutation that causes a condition called hypertrophic cardiomyopathy that can lead to heart failure and sudden death.

CRISPR-Cas9 uses guide-RNA molecules to target and cut specific segments of DNA.

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The objective: to cut the defective DNA to bring about its fix.

However, the new research, reported in the journal Nature, goes much further by modifying the nuclear DNA at the heart of the cell that influences personal characteristics such as height, facial appearance, eye colour and intelligence.

We all inherit two copies of each gene, one from dad and one from mom - and those embryos just copied the healthy one from the donated egg.

"The embryos are really looking for the blueprint", said Shoukhrat Mitalipov, who directs OHSU's Center for Embryonic Cell and Gene Therapy.

In more than half of the embryos, the DNA mutation was replaced with "healthy" DNA, and these embryos appeared to grow normally to the blastocyst stage (the point at which they would normally be transferred back into the woman's uterus during the IVF process - in this study, the blastocysts were destroyed during analysis).

A similar experiment had been conducted in China in 2015, but with mixed results. That is one of the technical problems that the latest experiments have overcome.

Scientists at the Oregon Health and Science University used CRISPR, a controversial, but easy to use, gene-editing tool to remove bad genes from DNA. "I don't think it's the start of clinical trials yet, but it does take it further than anyone has before". The work was consistent with recommendations issued this year by the National Academy of Sciences and the National Academy of Medicine joint panel on human genome editing. If not, "this technology will be shifted to unregulated areas, which shouldn't be happening", he explained. Because the embryos were created for research purposes only, none were allowed to develop beyond three days.

It may also help us to understand more about infertility, miscarriage and stillbirth, plus many diseases and disorders, by making better and new animal models of disease.

Asked about critics of genetic manipulation, he said his work has nothing to do with the creation of "designer babies" through genetic manipulation to give the children traits desired by the parents.

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