For first time, scientists edit DNA inside the body

Scientists took a pretty significant step this week when researchers edited a person’s DNA while it was still in his body.

The Associated Press reports the experiment was performed Monday in California on a man with Hunter Syndrome, which causes permanent, progressive damage affecting appearance, mental development, and organ function. There’s no cure, and people who have it are often dead by their teens. Until now. Maybe.

“We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending,” said Dr. Sandy Macrae, president of Sangamo Therapeutics, the California company testing this for two metabolic diseases and hemophilia. “It becomes part of your DNA and is there for the rest of your life.”

Think of the possibilities if it works. Think of the possibilities if it introduces another mistake to a person’s DNA.

“You’re really toying with Mother Nature” and the risks can’t be fully known, but the studies should move forward because these are incurable diseases, said one independent expert, Dr. Eric Topol of the Scripps Translational Science Institute in San Diego.

Protections are in place to help ensure safety, and animal tests were very encouraging, said Dr. Howard Kaufman, a Boston scientist on the National Institutes of Health panel that approved the studies.

He said gene editing’s promise is too great to ignore. “So far there’s been no evidence that this is going to be dangerous,” he said. “Now is not the time to get scared.”

Besides, there’s no cure and if a person figures he’s got nothing to lose, why not advance science?

“Many are in wheelchairs … dependent on their parents until they die,” Dr. Chester Whitley, a University of Minnesota genetics expert, tells the Associated Press. He’s planning to enroll patients in the study he’s leading.

The only alternative now to stem the progression of the disease is weekly doses of the missing enzyme that causes it. But that can cost $100,000 to $400,000 and it still won’t prevent the brain damage.

The description of the process used in the gene therapy is enough to make you proud to walk the same planet as the scientists who have figured out how to do it.

The therapy has three parts: The new gene and two zinc finger proteins. DNA instructions for each part are placed in a virus that’s been altered to not cause infection but to ferry them into cells. Billions of copies of these are given through a vein.

They travel to the liver, where cells use the instructions to make the zinc fingers and prepare the corrective gene. The fingers cut the DNA, allowing the new gene to slip in. The new gene then directs the cell to make the enzyme the patient lacked.

The potentially ground-breaking patient — it’ll be awhile before we know if the therapy worked — almost didn’t make it.

His flight to California was canceled on Sunday night and he couldn’t find another.

Science hasn’t come up with a cure for that yet.

  • MrE85
  • jon

    Love it when I hear about a long time science fiction staple becoming a part of reality…

    Now about those transporter beams…

    • It’s really amazing. Science is awesome. I still remember the first artificial heart transplant, and now look where we are.

      The human brain is at its best when it’s working for good.

      • Gary

        Sorry to be the advocate for the devil, but how long until someone says, “Hey you know that gene editing thing with the viruses, what if we put bad genes into those viruses and drop them on our enemies?”

        • Rob

          There’s probably a black op already underway.

        • How would you do that? And what would be the point?

          • Gary

            I agree science is awesome and best when working for good, but if there is a way to weaponize this, people are thinking about it. I don’t know how you would do it, but I have an idea why. It’s clean. Wipe out your enemies with disease, no bullets, no bombs, just a quick effective piece of DNA in a virus. Then move right in. Take over their land, take over their resources, and do it with their infrastructure still in place. I hope I’m long dead before this bit of science fiction were to ever become a reality.

          • RBHolb

            I recommend the work of Jacob Bronowski, especially The Ascent of Man. He made these remarks in one episode:

            It’s said that science will dehumanize people and turn them into numbers. That’s false, tragically false. Look for yourself. This is the concentration camp and crematorium at Auschwitz. This is where people were turned into numbers. Into this pond were flushed the ashes of some four million people. And that was not done by gas. It was done by arrogance, it was done by dogma, it was done by ignorance.

          • What you’re describing is more likely with biological warfare than gene warfare. If we’re planning on waging a war and are patient enough to wait generations for the widespread impact of an entire population of whatever characteristic it is that we consider victory, and we can get enough people to sit patiently for the IV, I guess that’s something that someone could do. But war is about killing as quickly and efficiently as possible and this would not be an efficient means to that end.

          • jon

            So your plans to weaponize this are slightly flawed…

            First off the method described above would require each and every one of your victims to allow you to inject them with the virus. (If your enemies allow you to stick them with a needle you have already won (should be straight out of the art of war)) that virus pay load doesn’t cause an infection…. That is to say it doesn’t reproduce, if it did there would be an immuno responses, and even if the victims immune system didn’t stop the virus, the reproduced copies wouldn’t likely not have a suitable transmission vector, not would they be equipped with the zinc for their next round of infecting…

            Second off, genetic manipulation while probably able to be fatal would take a while, and there is a chance unmodified cells would out produce modified ones (especially if the modified ones are sick and designed to die off, like you would want in something designed to kill people).

            Lastly, why go through the trouble? Ebola samples are easy to come by… If you have the skills to modify a virus to manipulate genes, you can probably do something to make a flesh eating virus more deadly, and transmission is already not an issue since nature took care of it for you.
            It’s like if you knew that a few shaped charges could turn plutonium into a nuke, but thought instead to build a fully compliant grid scale nuclear power plant, and then over ride all the safeties so you could cause it to melt down and release radio activity on people… Sure it’s doable, but an unshaped charge would have the same effect (dirty bomb) and a shaped charge would have had a far greater effect (fussion bomb), and some isotopes of hydrogen would have made the whole thing even more effective (fission bomb) but for some reason you opted to go through the most expensive process with the least effective outcome…

            Just because something can be done doesn’t mean it makes sense to be weaponized… We never weaponized sliced bread (well unless you listen to the anti gluten folks) because it would be a terrible weapon, and not particularly cost effective (burying your enemies in a lethal dose of bread just doesn’t make sense)… We have yet to weaponized mri’s, the inverse square law makes it impractical, we don’t weaponized blood transfusions, or heart transplants…

            We’ve got good cheap ways of killing each other easily available, no reason to create complicate inefficient and expensive ones… It’s just bad business.

        • MikeB

          There’s always a flip side to new discoveries and breakthroughs. It’s worth the price, and unavoidable.

  • Rob

    When the scientists did the editing, did they find any grammatical errors to correct?

  • Jeff

    I had to look up zinc fingers: https://en.wikipedia.org/wiki/Zinc_finger.

    The modular nature of the zinc finger motif allows for a large number of combinations of DNA and RNA sequences to be bound with high degree of affinity and specificity, and is therefore ideally suited for engineering protein that can be targeted to and bind specific DNA sequences.

    Apparently like chicken fingers they don’t necessarily contain zinc. The article didn’t specify what dipping sauces are best with them. (yuk, yuk)

    • RBHolb

      “Zinc Finger” was a book title Ian Fleming considered briefly, but ultimately rejected.

  • EarthToBobby

    Engineer me a pair of gills, please.

    • Rob

      Waterworld!

  • Guest

    Kudos to those who ADVANCE society……even if they are not paid as well as those who entertain society……sigh

    • ec99

      Sangamo stock went up 13.5% on the news.

  • Jack Ungerleider

    Whenever stories like this make the news, I long for the days when Art Kaplan was at the U of Mn and a regular visitor on Midday (or was it Midmorning). As with anything of this nature, lets hope the ethics catches up with the science quick enough to avoid potentially bad decisions on the use of technology like this.