Genome editing is a quickly-developing, game-changing field promising to influence the future of life on our planet.
Mapping the human genome has been a long and difficult process. Recently, sequencing technology has become more accessible and affordable to research labs, which can enable them to work towards personalized medical treatments for vexing diseases like cancer.
Advancements in genome editing will have a profound effect on all living things, potentially helping us to live longer, healthier lives.
Ten years ago, researchers unveiled a gene editing technique called CRISPR-Cas9, which allows scientists to edit precise positions on DNA using a bacterial enzyme. More recently, new technologies have made CRISPR gene editing more affordable. The implications are tremendous. Mosquitoes carrying malaria could be edited so that they no longer carry the disease through future generations, and so that millions of humans in high-risk regions are no longer exposed to the disease. There are therapeutic possibilities in human medicine as well. Editing our genetic code could mean eradicating certain genetic diseases—like cystic fibrosis—so they can’t be passed along to our babies, and liver cells could be edited to lower the bad cholesterol levels in families carrying inherited mutations. World-renowned geneticist George Church and his team used CRISPR to modify pig organs, making them safe to be used in human liver, kidney, heart and lung transplants.
This year, CRISPR will face a few key trials that will test its ability to treat genetic diseases and mitigate the growth of cancerous cells. In one trial, CRISPR will be used to disable genes in T-cells to help a cancer patient’s immune system effectively fight malignant cells from growing. It will also be used as a method of improving vision in people with an inherited condition that causes progressive blindness, and as a treatment for sickle cell disorder.
In 2015, Chinese researchers edited the genes of a human embryo. It was done in a petri dish, but scientists quickly sounded the alarm, because it wasn’t difficult to see how CRISPR could be used to modify embryos during the in vitro fertilization process. Since then, plenty of experimentation on human embryos has ensued in China, and late in 2018, we learned about a team of researchers led by Dr. He Jiankui at the Southern University of Science and Technology in Shenzhen who not only used CRISPR in conjunction with IVF, but purportedly eliminated the CCR5 gene in a pair of twin girls. That modification, the scientists hoped, would make the twins resistant to HIV, smallpox and cholera throughout their lives. If true, this would be the first instance of genetically modified humans—and we haven’t yet developed global norms and standards governing humanity’s position on this sort of human enhancement. In December 2019, Chinese state media revealed that Dr. He’s work had resulted in additional births beyond the twins. Authorities arrested him and sentenced him to three years in prison for “illegal medical practices.”
The revelations that a Chinese doctor had genetically edited human embryos that resulted in live births created a global panic. The scientific community lambasted Dr. He, while governments around the world were quick to publicly condemn the use of CRISPR for designer babies of any kind. Many nations, including the U.S., have regulations banning gene modification for that purpose. This will be a very difficult area to traverse heading into the future, especially with no global standards for modification. Genetic modification that aims to eradicate disease, however, could be a boon for humanity: Harvard University’s Stem Cell Institute started using CRISPR to modify sperm cells so that they could not pass on the genes responsible for Alzheimer’s disease. Back in 2017, the first known attempt to create a genetically-modified human embryo in the United States took place at the Oregon Health and Science University. Researchers there successfully corrected a genetic mutation causing a deadly heart condition. (The critical difference: These experiments did not result in pregnancy or live birth.)
Meanwhile, researchers at Stanford University have discovered that some people could be immune to part of the CRISPR process. One of the primary tools used, Cas9, is typically created using the same bacteria that causes strep throat. Some people’s immune systems can naturally fend off the infection, and this research calls into question whether the CRISPR technique could be effective across all—or just part—of the human population.
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