One microscopic step for man, one large step for mankind

August 9, 2017

Last Wednesday, the world got news of a breakthrough that will affect the future of not only medicine, but prenatal care as well: The first successful editing of human genes to repair a common and serious disease-causing mutation.

The process, gene editing, has been talked about for years. It’s been not only the center of scientific discussion, but also great fodder for science fiction as well as the center of many moral debates as to whether embracing the technology would change the nature of what a human being is.

So what actually happened?

Well, to put it in simple terms, the scientists went into a human embryo and removed a gene that could cause a potentially fatal heart condition.

The more complex explanation?

Using CRISPR-Cas9, the scientists entered the embryo right after fertilization, before it hit its eight-cell stage and removed a gene that causes hypertrophic cardiomyopathy, a disease, which leads to a thickening of the heart’s muscular wall. This disease, affects one in 500 people and is a common cause of sudden cardiac arrest in young people.

By removing the gene, it also means that the condition would not be passed to future generations. While genes have been edited before, this is the first time that the editing has not caused other mutations in the embryo.

By the way, the CRISPR-Cas9 is the name of a technology that edits gene. It is a system consisting of two key molecules that introduce a change into the DNA. These are an enzyme called Cas9, which acts as a pair of “molecular scissors” that can cut the two strands of DNA at a specific location, and guide RNA, which consists of a small piece of pre-designed RNA sequence located within a longer RNA scaffold.

Now the work that has been done could potentially be used to treat more than 10,000 inherited diseases. It’s been speculated that these could be used to remove genes that cause everything from breast and ovarian cancer to sickle cell anemia and cystic fibrosis.

Imagine how many less people would be dying each year and how many fewer families will be forced to say good-bye to a relative claimed early by a disease that couldn’t be stopped. Just imagine the relief on knowing that a disease that killed a love one, will not be passed on to your child or their children.

It sounds pretty promising.

It also, at least to me, sounds like a good thing.

But, unfortunately, there’s a fear that’s been created, largely by science fiction and modern medicine skeptics that the technology would be used to create “designer babies.”

“Designer babies” is pretty much the term applied to people choosing the traits they want in their kids. This could be stuff as simple as blond hair, green eyes or height to stuff like intelligence and musical talent — kind of like a eugenics program to create a genetic elite, much like the movie “Gattaca.”

Well, realistically, we’re not near that point yet. Many of those genes, as well as many genetic diseases, are multifactorial, which means they are caused by more than one set of genes. Multifactoral conditions include things like autism, Alzheimer’s and asthma.

Also, playing into this are the factors outside of the genes. Genes, are the blueprint, but ultimately, its the outside factors that determine things such as “What kind of environment do you live in?” “What do you eat?” nd “What do you like doing?”

You could be born with all the leg ups in the world, but it doesn’t matter much if you don’t use them.

This work with gene editing was a multi-country effort. The work was done by scientists not only in the U.S., but also South Korea and China. Like most breakthroughs, it was a cumulative work, built upon years and years of research. While many Americans picture China as a rival at best and a dangerous enemy at worst, it’s not unusual for us to work with countries that we have complicated relationships with on science.

Right now, we’re still years away from actually seeing gene editing become commonplace. When it is, expect it to be very regulated.

Anyway, we don’t have the ability to work on those genes yet. But, when we do, it’s natural for us to want to put rules into place. After all, we don’t want to say “forget the fatal heart condition — we can’t have people choose to have their kid born blond!”

Honestly, does that sound like a reason not to save lives?

Originally printed in the Batesville Daily Guard

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