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Technology explainer

CRISPR gene editing

A programmable molecular tool that cuts or changes DNA at a chosen address.

Maturity: Approved in some uses
Abstract editorial illustration of three parallel strands—one receiving a new segment, one being precisely snipped, one being dimmed—representing gene therapy, gene editing and RNA medicines.
Original illustration by Spin Pharma.

How it works

A guide RNA leads a Cas enzyme to a matching stretch of DNA, where it cuts. The cell’s own repair systems then disable a gene or, with newer base and prime editors, rewrite individual letters without a full double-strand break.

Why it matters

It can correct or bypass the cause of some inherited diseases in a single treatment. The first CRISPR-based medicine, Casgevy, was approved by the FDA in December 2023 for sickle cell disease.

Limits & open questions

Off-target edits, delivery to the right tissues, long-term safety follow-up, manufacturing complexity and very high costs all remain challenges. Current approved uses edit cells outside the body; in-body editing is still in trials.

Sources

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Stories about this technology

Abstract editorial illustration of a DNA double helix unspooling into lines of glowing letters, some highlighted and some faded to suggest known and unknown meaning.Genome + Cells
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