CRISPR Used To Edit Heart Defect Out of Human Embryos

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http://sci-hub.bz/10.1038/nature23305

http://archive.is/PqGCs

This is the first time gene editing on human embryos has been conducted in the United States. Researchers said in interviews this week that they consider their work very basic. The embryos were allowed to grow for only a few days and there was never any intention to implant them to create a pregnancy. But they also acknowledged that they will continue to move forward with the science with the ultimate goal of being able to “correct” disease-causing genes in embryos that will develop into babies.

The experiment is the latest example of how the laboratory tool known as CRISPR (or Clustered Regularly Interspaced Short Palindromic Repeats), a type of “molecular scissors,” is pushing the boundaries of our ability to manipulate life and has been received with both excitement and horror.

The most recent work is particularly sensitive because it involves changes to the germ line, that is, genes that could be passed on to future generations. The U.S. forbids the use of federal funds for embryo research and the Food and Drug Administration is prohibited from considering any clinical trials involving genetic modifications that can be inherited.

The research involved eggs from 12 healthy female donors and sperm from a male volunteer who carries the MYBPC3 gene which causes hypertrophic cardiomyopathy (HCM). HCM is a disease of the heart muscles that can be terrifying because it can cause no symptoms and remain undetected until it causes sudden cardiac death.

At around the time the sperm was injected into the eggs, researchers snipped out the gene that causes the disease. The result was far more successful than the researchers expected: As the embryo's cells began to divide and multiply, a huge number appeared to be repairing themselves by using the normal, non-mutated copy of the gene from the females' genetic material. In all, they saw that about 72 percent were corrected, a very high number.