OHSU Researchers 1st In US To Correct Genes With Editing Technology

First human embryos edited in the USA. Here's why it's problematic.

Having confirmed that the disease-causing mutation is repaired correctly in human embryos, IBS researchers performed further analysis to make sure that the gene scissors did not cut any other sites of the human genome.

The latest effort, led by Shoukhrat Mitalipov of Oregon Health & Science University, with researchers from South Korea, China, the Salk Institute for Biological Studies in California and others, involved editing the DNA of single-cell embryos with CRISPR-Cas9, a tool for genome engineering that is much simpler, faster and cheaper than earlier methods, and which has sparked an explosion of interest in possible applications. The genetic defect corrected in the study is the leading cause of sudden heart attack in young people, carried by about 1 in 500.

The new study focused on a hereditary genetic mutation that can cause hypertrophic cardiomyopathy, a relatively common and potentially life-threatening form of heart disease.

But the new study, published in Nature, suggests that CRISPR might work as an aid to fertility clinic screening for unsafe inherited diseases, relying on a previously unknown ability of human embryos to swiftly fix their genes.

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In the US, scientists can perform laboratory embryo research only with private, not federal taxpayer, funding, as the OR team did.

An worldwide group of 11 organizations, including the American Society of Human Genetics and Britain's Wellcome Trust, on Wednesday issued a policy statement recommending against genome editing that culminates in human implantation and pregnancy, while supporting publicly funded research into its potential clinical applications.

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

With strict regulation and an emphasis on ethical experimentation, the scientific breakthroughs that can be realized through these experiments could not only eradicate disorders like cystic fibrosis, muscular atrophy and even color blindness, but allow us to learn more about human development than ever before. Researchers worked with healthy egg cells donated by women and sperm from a man affected with hypertrophic cardiomyopathy, the IBS said. Based on prior research, 50% of the embryos in the study were expected to carry the bad gene. The research team cleverly side-stepped mosaicism by using CRISPR at the same time as fertilising the egg, before its cells had begun dividing.

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Additionally, we're at least decades away from having gene therapy techniques like this one become the norm.

The embryo studies were conducted in the U.S. where this procedure is legal, in adherence to guidelines established by OHSU's Institutional Review Board and additional ad-hoc committees established for scientific and ethical review. In the new study, researchers discarded the embryos a few days after fertilisation. An example of a legitimate issue regarding genome editing is whether the technology will be safe for clinical use in the near future. But the prospect of editing a child's DNA before they are born remains very controversial. And groups like the American Society of Human Genetics mostly center their opposition on modifying embryos for implantation into a human.

Echoing similar concerns, Fr. Pacholczyk pointing as well to the guidance from the National Academies of Sciences' 2017 report on human gene editing. Any attempt to study germline editing in pregnant women would require permission from the Food and Drug Administration, which is now prohibited by Congress from reviewing any such request.

"Although it implies a future in which we could choose to have children without particular genetic diseases, or even design for particular traits, that is still likely a very long time from now", he wrote in an email to KPBS.

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