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  • Writer's pictureMichael Goettler

The Potential and Perils of In-Utero Gene Editing


In-Utero

Beta-thalassemia, cystic fibrosis, hemophilia, sickle cell anemia…the list of genetic diseases goes on, but a cure is in short supply.


Recent advances in gene therapy are delivering potential cures to a select few. These ground-breaking innovations are incredible, bringing new life to patients with conditions like beta-thalassemia and sickle cell anemia who have suffered for years. However, treatment comes with substantial costs and a difficult and long-lasting treatment course.


What if gene therapies could be introduced in utero? Altering the single gene in question could eliminate the disease altogether, avoiding associated symptoms and treatments at a significantly lower cost.


The idea is not as farfetched as it seems. Examples of human and animal gene editing are already underway and in the news. Researchers recently made headlines for successfully using CRISPR-based editing in fetal monkeys, reducing toxic proteins that can cause fatal liver disease. The editing apparatus was found in other major monkey organs, showing that the genetic changes could reproduce widely.  


In the big picture, using science to prevent disease can be ethical when following standardized rules. But what are those rules and where do we draw the line? 


How do we navigate the slippery slope of gene editing, and how do we prevent its arbitrary use for non-vital applications like choosing “designer” traits for offspring?


One bad actor illustrates why this matters. A researcher in China made international news, after being imprisoned for violating medical regulations. He allegedly used CRISPR-Cas9 editing on human embryos which were then implanted (they are currently 5-year-old twins) to provide HIV immunity. This caused a rightful outcry for ignoring ethical concerns.


As we move forward with in-utero gene editing research, we must consider risks to the health of the developing fetus and the mother, as well as concerns over accidentally affecting the germline. That’s just the beginning of the list of considerations.


Where do we draw the line in embryonic and in-utero gene editing? Is it acceptable as long as it treats genetic diseases?

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