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The future of gene editing

By David Prentice, PhD | January 14, 2016

Excerpted from "Will Gene Editing Be in Your Medical Future?," Medscape. January 5, 2016 — Gene editing is a very compelling concept for physicians. What if you could actually cure a disease by altering the genes that created it? Then your patients wouldn't need drugs and other therapies, which often involve high costs and dangerous side effects. This revolutionary approach could either remove the disease or reduce it to a nonthreatening level.

Slowly but surely, researchers are trying to bring the concept of gene editing closer to clinical reality. Still, no one is saying that this therapy would be commercially available any time soon. Use of gene editing on humans is just beginning to enter clinical trials. At this point, research is focusing only on a small number of diseases that affect relatively small populations.

In gene editing, "the idea is not to treat the disease but to physically change the DNA in a way that cures the disease," says Fyodor Urnov, PhD, a genetic biologist and senior scientist at Sangamo BioSciences, a California company that owns the rights to a form of gene-editing technology called "zinc-finger nucleases."

More than 3000 diseases have been linked to mutations in individual genes, but researchers are starting with diseases that are most likely to yield positive results. These include HIV and diseases that involve a defect in only one gene, such as hemophilia, sickle cell disease, and beta thalassemia. Meanwhile, "there are many diseases that we are not looking at, such as heart disease, because they have contributions from multiple genes," Dr Urnov says.


Dr. David PrenticeCMDA Member and Research Director at Charlotte Lozier Institute David Prentice, PhD: “Gene editing will indeed be in our medical future. The possibility of correcting genetically-based problems and treating diseases based on altering genetic expression in cells and tissues holds significant promise for future medicine.


“As we contemplate that future, we must also consider how and when genetic treatments should be used. There is little controversy in the potential use of gene editing to treat pathologies of born or unborn patients, alleviating individuals of life-threatening and disabling conditions. Yet some have advocated application of genetic manipulation earlier, at the embryo stage, and experiments have already been done on human embryos to show proof-of-principle. While this genetic engineering supposedly would be done to ‘prevent’ an individual from carrying a genetic mutation, such genetic changes would not only affect the individual so manipulated, but would be passed through the germline to future generations, with unknown implications for all of humanity and not only the ‘designer babies.’

“Others propose extending genetic modification to non-disease traits, or even adding enhanced capabilities to the human genome; indeed, the concept of gene-doping has already been a concern at the Olympics. Congress recently added a funding amendment to pause the headlong rush toward genetic engineering of human embryos, to give more time for contemplation of our genetic future. We need to consider the patients who might benefit or be harmed, and we must also give thoughtful consideration to that ancient question: ‘What is man, that thou art mindful of him?’

Modest but Meaningful Protection from Human Embryo Genetic Manipulation by David Prentice, PhD
CMDA’s Human Enhancement Ethics Statement
Why the Church Needs Bioethics by John F. Kilner, Ph.D.

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