A Breakthrough in Gene Editing

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Written By Pinang Driod

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I spoke with my colleague Sarah Zhang about a breakthrough in CRISPR therapy, and when it is ethical to use the gene-editing technology.

First, here are three new stories from The Atlantic:

  • My father, my faith, and Donald Trump
  • Why the Fifth Circuit keeps making such outlandish decisions
  • Substack has a Nazi problem.

A Transformative Treatment

Earlier this month, U.K. regulators approved a new therapy that uses CRISPR—a gene-editing technology that allows scientists to make cuts to DNA—to treat people with sickle-cell disease. FDA approval is likely in the coming weeks. I spoke with my colleague Sarah Zhang, who has been covering CRISPR for more than a decade, about this landmark treatment, ethical use of the technology, and what fair access to CRISPR therapy could look like in the future.

Lora Kelley: What makes sickle-cell disease an obvious match for CRISPR therapy?

Sarah Zhang: As soon as scientists started talking about what we can do with CRISPR, treating sickle cell rose to the top of the list for two reasons. One is that with sickle-cell disease, you can take the blood cells out, edit them in the controlled environment of a lab, and then put them back in someone’s body. And with sickle cell, we know exactly the edit to make to treat the disease. For patients who have gotten the therapy, it’s been transformative. They’ve gone from being hospitalized multiple times a year to having virtually no symptoms.

Lora: Where does the scientific community draw ethical lines on when to use CRISPR?

Sarah: If you’ve heard about CRISPR and humans, you probably remember the CRISPR babies that were born back in 2018. A Chinese scientist went rogue and edited embryos that were then born as twins in China, purportedly to make them resistant to HIV.

CRISPR babies were an unnecessary and reckless use of gene editing. That moment catalyzed the community to think about how we want this technology to be used.

After that, there was a widely accepted consensus among scientists: no editing in sperm, eggs, or embryos. Edits in the DNA of your blood cells or muscle cells or brain cells don’t get passed on if you have children, the way they would be in sperm, eggs, or embryos. The rewards are also not clear. There aren’t really good applications where you could prevent a genetic disease from being inherited with gene editing that you can’t already do using IVF and embryo selection.

But everyone I talked with feels like this sickle-cell treatment is an appropriate use of CRISPR. The big question going forward is: Who can actually get access to this therapy?

Lora: What would equitable and fair access to CRISPR therapy to treat sickle-cell disease look like?

Sarah: This therapy is likely to be very expensive. It may cost around $2 million a person. It’s also hard to get physically. You’re basically doing a bone-marrow transplant on yourself. Your blood cells are taken out of you and edited; meanwhile, you’re undergoing chemotherapy to kill your remaining bone marrow. And then you’re getting your edited cells infused back into you, and rebuilding your blood and immune system over the course of several months.

So you have to be either in the hospital or going to the hospital and going to see doctors for about a year. If you live near one of these transplant centers, you may be able to do that. But if you live in a rural location, or maybe you are in school, or you have kids, or you have a job, you can’t take a year out of your life to undergo this therapy. Most people who have sickle-cell disease live in developing countries, largely in sub-Saharan Africa. So this therapy is logistically unfeasible for them.

Still, there’s some amount of justice to the fact that this groundbreaking therapy is helping treat a disease that predominantly affects Black people, who have been historically—and still are—marginalized in the medical system. The idea is that one day this treatment can be something more like a shot, which would be cheaper and easier to get.

Lora: How do you anticipate CRISPR being used to treat diseases in the future?

Sarah: The next step will be: How do we treat CRISPR right in the body? We’re starting to do that. There was a recent trial to lower people’s cholesterol using CRISPR. Lipid nanoparticles were used to send CRISPR to the liver, which is a relatively easy target. Changing things in the brain, the heart, or muscles is a lot harder. A big question now is: How do we get CRISPR to the cells that we want to edit?

Even though CRISPR has been described as a very precise gene editor, it’s actually still quite limited. It’s not the same thing as opening up Google Docs and changing some letters around. But now there are new technologies where you can change a single letter, or paste in a sequence, and that will allow for much more precise edits in the future. CRISPR is quite easy to use. But making sure you do all the right edits is harder.

Still, these breakthroughs have been exciting for me personally. I started covering CRISPR very early in my career, when it was a thing you did to cells in a petri dish in a lab. Now it’s being used to treat humans. It’s only been a little bit more than 10 years, which feels like a long time, but in the medical world, it’s really a blink of an eye. It’s extraordinary that it happened so quickly.

Related Links

  • The CRISPR era is here.
  • CRISPR has a terrible name.

Today’s News

  1. Forty-one workers were rescued after a tunnel collapsed in the Indian state of Uttarakhand; they had been trapped for more than two weeks.
  2. Americans for Prosperity Action, a political network founded by the billionaire brothers Charles and David Koch, has endorsed Nikki Haley in the Republican primary for president.   
  3. Israel and Hamas will extend a temporary pause in fighting until tomorrow, maintaining the possibility of further extensions and hostage-prisoner exchanges.

Evening Read

Painting by Fulton Leroy Washington (MR WASH). Source: Malike Sidibe for The Atlantic.

This Is Not Justice

By Jake Tapper

Editor’s Note: As of yesterday, C. J. Rice, the subject of our November 2022 cover story, could be very close to freedom after a federal court overturned his conviction. The Philadelphia District Attorney’s Office now has 179 days to decide whether to retry Rice’s case or release him from custody. Read our update here.

On Tuesday, September 20, 2011, a young patient walked haltingly into a medical office in South Philadelphia to have his bullet wounds examined.

The patient was a 17-year-old named C. J. Rice, who lived in the neighborhood. The doctor was a pediatrician named Theodore Tapper.

My father had been working as a physician in South Philadelphia for more than four decades and had known Rice since he was a child. Rice had been brought in for a checkup soon after he was born, and as a doctor my father had seen Rice several times a year, along with other members of the family. Two weeks and three days before his September appointment, Rice had been shot while riding his bike, in what he believed was a case of mistaken identity. To remove one of the bullets, a surgeon had made a long incision down the middle of Rice’s torso. The wound was then closed with a ridge of staples—more than two dozen. After his discharge, Rice was in severe pain and could barely walk. He needed help to get dressed in the morning and help to go up and down stairs …

The timing of that visit is significant because, six days later, the Philadelphia police announced that they were seeking Rice and a friend of his, Tyler Linder, in connection with a shooting that had occurred in South Philadelphia on the evening of September 25 and left four people wounded, including a 6-year-old girl.

Read the full article.

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Culture Break

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Katherine Hu contributed to this newsletter.

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