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prensaestatal.com Octubre 23, 2017


First embryo gene-repair holds promise for inherited disease

03 Agosto 2017, 05:40 | Martinez Canez

Image Shutterstock

Image Shutterstock

Genome editing was singled out for concern in a 2016 report to Congress from the us intelligence community about potential wordwide threats: "Given the broad distribution, low cost, and accelerated pace of development of this dual-use technology, its deliberate or unintentional misuse might lead to far-reaching economic and national security implications".

In their new paper, a consortium of scientists in California, Oregon and Asia detailed using the genome-editing technique CRISPR to fix DNA that causes a common genetic heart disease known as hypertrophic cardiomyopathy.

The Ministry of Science and ICT said on Thursday that the researchers from the Institute for Basic Science (IBS) and Oregon Health and Science University (OHSU) used a new gene-editing technique and succeeded in correcting the mutated gene in human embryos which causes the heart disorder called hypertrophic cardiomyopathy. "Clinical trials would mean actually implanting some of these embryos with the goal of establishing pregnancy and monitoring births of children and hopefully following up with children".

The researchers said more work is needed to flawless the technique and see how widely it could be applied. "I was told at age 12 that I could die suddenly and that there was nothing anyone could do about it", she says. "And we need more basic research like this before we can even consider going forward".

Nature stepped in at this point in a clever way, by using the egg donor's properly functioning MYBPC3 gene as a template on which to base the fix. The condition causes deadly heart attacks in otherwise healthy young people. Work with skin cells reprogrammed to mimic embryos had suggested the mutation would be repaired in fewer than 30 percent of cells.

In one embryo, the researchers did attempt to insert new DNA, but that embryo experience mosaicism. So in order to further the science, Mitalipov and his colleagues wanted to test what happened when CRISPR was used in a human embryo.

Reassuringly, they didn't find the same level of off target effects other studies have experienced.

The first gene editing of a human embryo is a remarkable breakthrough in genetic editing science, although there remains some controversy surrounding the subject. He and Kahn were part of a National Academy of Sciences report earlier this year that said if germline editing ever were allowed, it should be only for serious diseases with no good alternatives and done with strict oversight.

Researchers also are using gene editing to hatch malaria-resistant mosquitoes, grow strains of algae that produce biofuels, improve crop growth, even make mushrooms that don't brown as quickly.

Wu said timing was key for preventing off-target effects and mosaicism.

"We don't like the word editing because we didn't edit anything".

The team also uncovered a new and potentially important DNA fix mechanism that takes place in early embryo development.

The new procedure tackled a genetic mutation in human embryos that causes hypertrophic cardiomyopathy, an inherited condition in which the heart muscle becomes abnormally thick.

Because these edits would not only affect that child, but could also be passed down to their descendants, some ethicists are firmly opposed to editing of the human "germline". The first two of those studies used defective IVF embryos that could never develop into a baby (they had been inadvertently fertilised with two sperm) as a way to sidestep the ethical minefield.

Other strategies now exist for preventing genetic diseases in offspring.

The work has impressed other scientists in the field because in previous experiments, gene editing has worked only partially, mending harmful mutations in some cells, but not others. Such tinkering with embryo DNA, called germline editing, is controversial because of fears that the technology will be used to create so-called designer babies. "The technology is not there yet". What kinds of genes should we consider "ethical" to correct? The second study, published in 2016, edited a gene to confer HIV resistance to the embryo.

University of Wisconsin-Madison bioethicist Alta Charo thinks that the benefits of this potential treatment outweigh all concerns. "By using this technique, it's possible to reduce the burden of this heritable disease on the family and eventually the human population", Mitalipov says.

Michael Kalichman, director of the Research Ethics Program at UC San Diego, agreed that the study did not signal that parents will soon have the option to pick and choose from a menu of desired traits for their offspring. In the absence of editing, and had they been allowed to develop, they would have grown into adults likely to suffer from the disease.

"This technology can theoretically be applied to any other heterozygous mutation", Amato says.



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