Tag Archives: gene editing

“Scientific Breakthrough:” Editing Genes in Human Embryos

Publications have been reporting on a recent breakthrough in modern medicine: modifying human DNA in human embryos, without introducing the critically serious disease-causing mutations that were problematic in previous attempts.

Published online Wednesday in the journal Nature, the research is targeted towards assisting families with genetic diseases. The new research experiment utilized a gene-editing technique to correct a genetic defect behind a heart disorder, that can cause “seemingly healthy young people” to die from sudden heart failure. Scientists at Oregon Health and Science University, in conjunction with colleagues in California, China, and South Korea, reported that dozens of embryos were repaired: if those embryos developed, not only would they be disease-free, but also would not transmit the disease to future generations.

This is the first time that scientists have successfully edited genes in human embryos to prevent dangerous disease mutations. Scientists collectively agree that while the research is a major milestone and achievement, the prospect of human genetic engineering has already raised ethical concerns. Mary Darnovsky, director of the Center for Genetics and Society—a watchdog group based in Berkeley—says that it is “a flagrant disregard of calls for a broad societal consensus in decisions about a really momentous technology that could be used for good, but in this case is being used in preparation for an extraordinarily risky application.”

Yet the researchers emphasize that the work is focused on preventing debilitating diseases and disorders, not the creation of genetically enhanced babies. Richard Hynes, a cancer researcher at the Massachusetts Institute of Technology who co-led the committee, says that their report sought to eliminate the technical hurdles, but there will inevitably be “societal issues that have to be considered and discussions that are going to have to happen.”

While the overwhelming consensus is that much more research is required before the method could be tested through clinical trials—which is currently not permissible under federal law—the technique could potentially apply to over 10,000 conditions caused by specific inherited mutations. This includes diseases like Tay-Sachs, Huntington’s, sickle cell anemia, cystic fibrosis, and many others.

Nevertheless, any scientists hoping to continue the work in the U.S. are presented with a host of regulatory obstacles. The research was specifically funded by Oregon Health and Science University, the Institute for Basic Science in South Korea, and a number of foundations, as the National Institutes of Health does not fund any work involving human embryos. Moreover, Congress has prohibited the Food and Drug Administration to consider any research or experiments that involve genetically modified human embryos.

Scientists in Britain have received approval to use CRISPR—the gene editing technology—to edit the DNA in healthy human embryos, in order to further research surrounding normal human development; a team in Sweden has also started similar experiments. Fredrik Lanner, a geneticist at the Karolinska Institute in Stockholm—conductor of the experiments—says that this needs to be highly regulated. “This is very exciting. But it also could be a double-edged sword. So I think we really have to be extra cautious with this technology.”

Human Gene Editing

The National Academy of Sciences, in collaboration with the National Academy of Medicine, has lent their support to a “once-unthinkable” proposition: modifying human embryos, in order to create genetic traits that can be transferred to future generations.

Notwithstanding the medical and scientific complications, human gene editing has historically posed an ethical dilemma, as scientists fear that techniques used to prevent hereditary and genetic disease might also be used to create specific physical traits, or enhance intelligence.

A special science advisory group has solely endorsed alternations that are designed to prevent babies from acquiring genes known to cause “serious diseases and disability,” and only when there is no “reasonable alternative.” This engineering might allow people to have children without fearing that they have passed on genetic traits for diseases and disabilities like Huntington’s and Tay-Sachs.

The advent of a specific gene-editing tool called Crispr-Cas9 has allowed researchers to alter, insert, and delete genetic material with rapidly increasing precision, and has spurred plans for experimental treatments of adult patients with conditions like cancer and blindness.

Yet opponents of this new technology argue that human gene line editing will lead to the engineering of traits like beauty, strength, intelligence—ultimately leading to the possibility of a disproportionate divide between those who can afford enhancements, and those who cannot.

There are also questions of safety and autonomy, in addition to social justice and moral concerns. Despite the precision of Crispr, its ‘off-target’ effects include cutting DNA at places it is not meant to—leading to the inadvertent creation of new complications. Furthermore, while the published report prohibited any alternations that resembled ‘enhancement,’ it is not clear where the line is drawn.

Nevertheless, it will likely be years before gene-editing techniques tested in animals can simultaneously work in humans. The Food and Drug Administration currently prohibits the allotment of any federal money to support research that results in genetically modified offspring. This groundbreaking step will likely only be considered and implemented after more research and studies, and only conducted under extremely tight restrictions.