CRISPR used in human trials for first time in U.S.

Do scientists know enough about gene editing to move forward with human trials?

  • Doctors used the gene-editing tool in an attempt to treat a 34-year-old patient with sickle cell disease.
  • Last year, a Chinese scientist caused major controversy when he used CRISPR to genetically edit two human embryos.
  • It's unclear exactly what risks are involved in gene editing.

For the first time in the U.S., doctors used the gene-editing tool CRISPR to alter the genes of a human.

The patient was Victoria Gray, 34, who suffers from sickle cell disease, a genetic defect in which bone marrow produces a defective protein that renders blood cells unable to carry oxygen effectively.

"It's horrible," Gray told NPR. "When you can't walk or lift up a spoon to feed yourself, it gets real hard."

In an experimental procedure, doctors took stem cells from Gray's own bone marrow and used CRISPR to disable the faulty gene — named BCL11A. This disabling boosts the production of fetal hemoglobin, an oxygen transport protein. All humans produce fetal hemoglobin when young, and for decades it's been thought that this protein, in particular, helps treat sickle cell disease.

Now, researchers are able to put that theory to the test.

"It's exciting to see that we might be on the cusp of a highly effective therapy for patients with sickle cell," Dr. David Altshuler, executive vice president, global research and chief scientific officer at Vertex Pharmaceuticals, which is conducting a study with CRISPR Therapeutics, told NPR. "People with sickle cell disease have been waiting a long time for therapies that just let them live a normal life."

Still, it's unclear what risks gene editing poses to humans, given that almost all past experiments have been limited to animals and culture dishes. One major risk factor has to do with the interconnectedness of genes and their unmapped functions. For example, in June, a paper published in Nature Medicine described how editing genes to yield one specific benefit might result in a number of unforeseen consequences.

Why CRISPR Gene Editing Gives Its Creator Nightmares

Specifically, the researchers of the new study were examining the potential consequences of a highly controversial gene-editing procedure conducted last year by a Chinese scientist named He Jiankui, who used CRISPR to edit two human embryos, giving them a genetic mutation that theoretically rendered them unable to contract HIV. The scientists' main takeaway? Their research suggests that people born with that specific mutation tend to die sooner than those who don't.

"There might be a public perception that one mutation does one thing, but biology doesn't work like that," Rasmus Nielsen, an evolutionary biologist who coauthored the paper with one of his postdocs, Xinzhu Wei, told Wired. "I hope this raises awareness that before we think about modifying the human genome with CRISPR, we carefully consider all the possible effects of the mutations that we create."

Still, for people with life-shortening diseases like sickle cell, the risks of gene editing might be well worth it.

"This gives me hope if it gives me nothing else," Gray told NPR. It will likely take years of further testing before gene-editing procedures could become available to a wider pool of sickle cell patients.

Befriend your ideological opposite. It’s fun.

Step inside the unlikely friendship of a former ACLU president and an ultra-conservative Supreme Court Justice.

Sponsored by Charles Koch Foundation
  • Former president of the ACLU Nadine Strossen and Supreme Court Justice Antonin Scalia were unlikely friends. They debated each other at events all over the world, and because of that developed a deep and rewarding friendship – despite their immense differences.
  • Scalia, a famous conservative, was invited to circles that were not his "home territory", such as the ACLU, to debate his views. Here, Strossen expresses her gratitude and respect for his commitment to the exchange of ideas.
  • "It's really sad that people seem to think that if you disagree with somebody on some issues you can't be mutually respectful, you can't enjoy each other's company, you can't learn from each other and grow in yourself," says Strossen.
  • The opinions expressed in this video do not necessarily reflect the views of the Charles Koch Foundation, which encourages the expression of diverse viewpoints within a culture of civil discourse and mutual respect.
Keep reading Show less

Physicists find new state of matter that can supercharge technology

Scientists make an important discovery for the future of computing.

Surprising Science
  • Researchers find a new state of matter called "topological superconductivity".
  • The state can lead to important advancements in quantum computing.
  • Utilizing special particles that emerge during this state can lead to error-free data storage and blazing calculation speed.
Keep reading Show less

Physicist advances a radical theory of gravity

Erik Verlinde has been compared to Einstein for completely rethinking the nature of gravity.

Photo by Willeke Duijvekam
Surprising Science
  • The Dutch physicist Erik Verlinde's hypothesis describes gravity as an "emergent" force not fundamental.
  • The scientist thinks his ideas describe the universe better than existing models, without resorting to "dark matter".
  • While some question his previous papers, Verlinde is reworking his ideas as a full-fledged theory.
Keep reading Show less

How to heal trauma with meaning: A case study in emotional evolution

As tempting as it may be to run away from emotionally-difficult situations, it's important we confront them head-on.

  • Impossible-sounding things are possible in hospitals — however, there are times when we hit dead ends. In these moments, it's important to not run away, but to confront what's happening head-on.
  • For a lot of us, one of the ways to give meaning to terrible moments is to see what you can learn from them.
  • Sometimes certain information can "flood" us in ways that aren't helpful, and it's important to figure out what types of data you are able to take in — process — at certain times.
Keep reading Show less