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CRISPR vs. Hypertophic Cardiomyopathy: Embryo Gets Clean Genes

04 August 2017
CRISPR vs. Hypertophic Cardiomyopathy: Embryo Gets Clean Genes

The Oregon Health & Science University collaborated with the Salk Institute and Korea's Institute for Basic Science to use a technique known as CRISPR-Cas9, which has been used in China with mixed results.

"I for one believe, and this paper supports the view, that ultimately gene editing of human embryos can be made safe". This technique would only work in the second case.

Will we see more USA experiments using CRISPR after these initial results? In the remaining 27.6% embryos, the cellular cut-repairing mechanism introduced some unwanted insertions or deletions near the cut.

Last week, reports circulated that doctors had successfully edited a gene in a human embryo - the first time such a thing had been done in the United States.

So far guidelines for using CRISPR are minimal...

Yesterday (Wednesday), it was revealed that for the first time ever, scientists succeeded in correcting flawed DNA of a human embryo, thus preventing it from developing hereditary heart disease. If even one cell in an early embryo is unedited, "that's going to screw up the whole process", says Mitalipov. "Whilst we are just beginning to understand the complexity of genetic disease, gene-editing will likely become more acceptable when its potential benefits, both to individuals and to the broader society, exceeds its risks". However, according to MIT Technology Review, the experiment was just an exercise in science - the embryos were not allowed to develop for more than a few days and were never meant to be implanted into a womb. Several days later, 72% of the embryos showed no sign of the mutated gene; the gene was essentially corrected in all of their cells. Then they analyzed all the cells in the early embryos at single-cell resolution to see how effectively the mutation was repaired. Furthermore, by cutting out a gene that caused a heart defect, the disease (called hypertrophic cardiomyopathy) would theoretically also be prevented from being passed down from generation to generation.

But there is also the prospect of avoiding heritable, genetic diseases that can handicap or kill. The genetic defect can lead to heart failure and death. Not only does it require embryos to be fertilized in a nontraditional, invasive - and likely expensive - manner, US regulations still bar this type of experimentation from resulting in a pregnancy. But that may require several cycles of IVF, which is expensive and carries with it side effects and complications, before enough genetically healthy embryos are created. The results of that initial study have now been released and the findings are quite astonishing. But if the technique is found to work safely with this and other mutations, it might help some couples who could not otherwise have healthy children. Researchers have improved precision in recent years, but out-of-body treatments like using cells as drugs get around the fear of fixing one problem only to spark another. The Salk scientists contributed by developing the gene editing strategy, initially testing it in stem cells derived from the patient's skin cells. Crispr, although it appears to be very precisely targeted, can make errors analogous to a badly framed search-and-replace request, changing gene sequences that are far from the intended targets.

While many are hailing the research as an exciting breakthrough, Dr David King of Human Genetics Alert in London told RT it could eventually lead to a "eugenics scenario".

Humanity has an ethical question it's going to have to think about now with today's announcement that scientists in OR have successfully edited genes in human embryos to fix a disease-causing mutation that often kills young athletes. The Committee on Human Gene Editing recommended a series of general values: transparency, wellbeing of people, fairness, and responsible science, in an age of DNA manipulation. Had they lived, the babies would no longer develop the heart condition or pass it on to their own children.