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November 23, 2023

CRISPR gene editing has arrived. Here’s what that means

The world’s first medical treatment using the CRISPR gene-editing tool has won approval. The implications are enormous.

In the news

The UK last week approved the world’s first medical treatment using the revolutionary gene-editing tool known as CRISPR. Because CRISPR (clustered regularly interspaced short palindromic repeats) enables precise changes to DNA, it has long been expected to have a major impact on life sciences and healthcare.

The approval of the treatment, called Casgevy, thrilled the science world because it represents a step forward for CRISPR therapy. In fact, observers say the US Food and Drug Administration appears poised to follow the lead of the UK and approve Casgevy (generic name: exa-cel) within the next few weeks.

Casgevy is intended to treat sickle cell disease and beta thalassemia, genetic conditions that cause debilitating pain. Previously, a bone marrow transplant was the only permanent treatment; in trials, the Casgevy gene editing treatment has been found to relieve the diseases’ symptoms by restoring healthy hemoglobin production.

The Casgevy treatment actually edits the faulty genes that cause sickle cell and beta thalassemia. Stem cells are removed by clinicians from patients’ bone marrow, after which CRISPR is used to alter the gene that, among sufferers of the diseases, prevents the production of beneficial fetal hemoglobin. Before the gene-edited cells are reinfused in the patient’s body, the patient goes through treatment to prepare their bone marrow.

These developments could accelerate the use of CRISPR for medical treatments and its potential to change medicine.

The Cognizant take

CRISPR, which earned discoverers Emmanuelle Charpentier and Jennifer A. Doudna a Nobel Prize in 2020, has been likened to “genetic scissors” due to its ability to manipulate DNA. Its potential is boundless. In agriculture, gene editing is being explored to increase crop resilience in extreme climates. In one exciting experiment, CRISPR was used to restore the vision of blind mice.

But it is in human beings that the potential—and potential perils—of CRISPR really show up. As illustrated by Casgevy, gene editing can repair the body in ways long thought unimaginable. But it could also theoretically be used to enhance traits thought desirable, which raises the alarming specter of a race of genetically tuned uber-humans.

The technology is also expensive and difficult to deliver, which raises equity issues. Nations and regions whose populations could benefit most from gene editing will likely be the last to see it implemented.

These issues bear watching and discussion at the highest level, but the ability to treat disease and sickness at the DNA level is one of the most thrilling developments in recent history.

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