Gene editing may seem like something out of a futuristic sci-fi movie to most of us, but the process has actually been around for decades; particularly in the livestock, farming, and agriculture industries. Though Gregor Mendel may have imagined a more sophisticated future for his hybrid pea plants, no one could have predicted the slicing and splicing that DNA researchers are now capable of. Recent breakthroughs have allowed for the sterilization of malaria-carrying mosquitoes, and produced therapies for spinal cord injuries, sickle cell anemia, and now, perhaps, heart disease.
In 2018, the University of Pennsylvania School of Medicine published their findings involving one gene mutation that naturally occurs in about 20% of humans. These findings show how researchers already knew that mutation would protect its carriers from arterial plaques that can lead to heart disease. However, many have wondered how to give the other 80% of humans the same advantage. In the UPenn study, the test subjects (mice), underwent gene editing to introduce the mutation with favorable results. The gene-edited mice had markedly lower cholesterol over the course of just one week. Also good news, the mice did not appear to have any side effects from the therapy.
Clear Arteries, Full Hearts, Can’t Lose
At the same time that cholesterol tests were being conducted on mice, a similar study was being done on much larger animals. In 2020, Dr. Sekar Kathiresan, with Verve Therapeutics, shared a summary of his team’s findings at the International Society for Stem Cell Research’s virtual conference. Kathiresan’s team applied similar gene editing tequniques like the ones used in the University of Pennsylvania mice study to thirteen monkeys. The results were even more dramatic. According to The New York Times, “Not only did the [treatment] work… but it appeared that every liver cell was edited,” meaning that the gene-editing had been adapted by newly generated cells. After the gene editing, the monkeys’ LDL levels dropped by fifty-nine percent within two weeks, and tryglyceride levels declined by sixty-four percent.
So what does this mean for human beings with high cholesterol? According to the University of Pennsylvania’s Kiran Musunuru, lead researcher on the mouse study, “This proof-of-principle study showed that base-editing of [Angiopoietin-like protein 3] is a potential way to permanently treat patients with harmful blood lipid levels, especially those with genetic disorders that can cause sky-high cholesterol levels and dramatically increase the risk of a heart attack.” Musunuru suggested that a complete therapy may be available in as few as five years.
Brown Eyes Or Blue?
Gene therapies, from genetically modified crops to cloning, have come with their fair share of controversies, though the promise of life-changing treatments has always been the carrot at the end of the stick. Diseases and disorders like Alzheimer’s, multiple sclerosis, ALS, diabetes, and even certain cancers have responded well to genetic manipulation. For now, however, these therapies can be cost-prohibitive or even illegal, depending on where the patient resides. For example, the United States has far stricter guidelines on its human tissue research and therapies than other nations. Outside of the US, some nations have come under fire for their go-for-broke approach to their gene experiments. For example, the cloning of different species and the insertion of human genes into the organs, including brains, of laboratory animals has been extremely controversial in the global medical community. Still, in some places, these studies do take place.
These nations feel that robust research and treatment related to gene editing is essentual; although they walk the fine line between ethical concerns and scientific innovation. Recent reports have highlighted gene-editing studies meant to solve blindness, cystic fibrosis, HIV, heart failure, glaucoma, and more.
Critics on gene therapies often accuse scientists of “playing God,” in the worst sense. Additionally, members of communities who are afflicted by the conditions scientists are trying to cure, like the deaf community, have expressed their view that gene editing is a means of erasing their very existence.
No matter the person, there is certainly a myriad of different opinions on the topic of gene editing. One can only hope that all voices are considered, and that decisions are made with the utmost care, as gene therapies will only increase in the future.
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