United States scientists edit out disease from human embryos for first time

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

Scientists at the Salk Institute in collaboration with colleagues in OR and South Korea have succeeded in correcting a disease-causing mutation in human embryos through gene editing. The scientists' intervention raised that rate to 72 percent.

It's the first time a mutation that causes a common inherited disease has been fixed using a gene-editing tool.

The scientists said the procedure was surprisingly effective and that clinical trials could eventually be considered.

"Looking at it more closely, it's less useful than you might expect, if it works at all", Greely said.

In the world of clinical trials, testing on an animal subject is the step before clinical trials on humans.

The researchers broke the mutated gene using a technology called CRISPR-Cas9. "The embryo did the fix".

Mitalipov said he hoped the technique could one day be applied to a wide variety of genetic diseases and that one of the team's next targets may be the BRCA gene mutation, which is associated with breast cancer.

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.

Although impressed by the advance made by Mitalipov and colleagues, experts feel the technique is far from being ready for clinical use and that preimplantation genetic diagnosis is still the best approach to preventing the transmission of serious genetically inherited diseases.

Gene editing is controversial because while it could be used to avoid deadly or disabling inheritable diseases, opponents argue it could theoretically be used to create "designer babies" with specific features deemed desirable. "It seems to be working from these samples that we have chosen, but we need to do much more basic research with many other genes".

The sperm came from a donor with hypertrophic cardiomyopathy.

"I don't want to be negative with our own discoveries, but it is important to inform the public of what this means", he said. Sometimes eggs had already copied DNA, and a mutant gene escaped editing (top, middle). As the embryo's cells began to multiply, a high number of them appeared to correct themselves using the nonmutated genes.

At the same time, the breakthrough research has reignited long-running debate over the ethics of artificially modifying human reproductive cells, including whether it should be practiced at all. In this case, 42 out of the 58 edited embryos were free of the mutation that causes hypertrophic cardiomyopathy.

Scientists have successfully managed to alter DNA in defective human embryos in order to remove a genetic mutation, in a groundbreaking move that could open the door to preventing some 10,000 inherited disorders in future.

Daley worries that the success reported in the paper could motivate a clinician to try the technique before it has been fully tested.

Once the defective elements of the gene had been snipped away, the embryo's own cellular fix systems replaced them with healthy versions. Salberg can name six relatives, including a 19-year-old great uncle and 36-year-old sister, that died early from the disease. The MYBPC3 mutation, in particular, was predicted to produce relatively few opportunities for off-target cutting. "If you told me we could go back to 1967 and my mother could do CRISPR editing to take out the [hypertrophic cardiomyopathy] mutation so I wouldn't have it, I'd have a very different life in so many ways". In a 2015 statement regarding its policy against funding research on gene-editing in human embryos, the National Institutes of Health said germline editing "has been viewed nearly universally as a line that should not be crossed". "It's unclear at this point when we would be able to move on", Mitalipov says. Should we take the risk of proceeding with the first full term human pregnancy, not knowing if the technology will have unexpected adverse consequences? That could limit the use of the method for introducing any sort of "designer" gene. For one, the efficiency of the CRISPR and fix process is still about 70%. Typically, researchers wishing to edit a genome will insert DNA encoding CRISPR components into cells, and then rely on the cells' machinery to generate the necessary proteins and RNA.

Reporter Shamini Bundell investigates a new development in the gene editing of human embyos.

In this case, scientists can analyze the embryos in vitro to weed out any with the genetic defect before implantation into the womb.

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