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UpdatedChanged2023

Gene editing is experimental, with no approved therapy

The core idea stands, but key numbers or details have changed.

TaughtCRISPR is a remarkable laboratory tool for editing DNA, but using it to treat patients is years away and purely experimental. No gene-editing therapy has been approved for use in people.

NowOn 8 December 2023 the US FDA approved Casgevy, a CRISPR-based therapy for sickle cell disease, the first approved medicine that edits a patient's DNA. The UK had authorized it weeks earlier.

What actually happened

CRISPR-Cas9 as a precise gene-editing method dates to 2012, and Jennifer Doudna and Emmanuelle Charpentier won the 2020 Nobel Prize in Chemistry for it. The leap from "we can edit DNA in a dish" to "this is an approved treatment you can receive in a hospital" took eleven years.

Casgevy treats sickle cell disease, a painful inherited blood disorder, by editing a patient's own blood stem cells to switch on fetal hemoglobin, which the sickle mutation does not affect. In trials it eliminated the debilitating pain crises that define the disease for the large majority of patients. It is a one-time treatment, and it is extraordinarily expensive, but it is real and approved.

Textbooks written even a year or two earlier framed gene editing as the promising future of medicine. As of December 2023 it is also the present. The word "experimental" quietly came off the label.

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Sources

  1. FDA: FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Diseasefda.gov
  2. Vertex and CRISPR Therapeutics: US FDA Approval of CASGEVYir.crisprtx.com

Who was taught this

Still standard through 2023, so anyone who finished school between 1950 and 2023 learned the earlier version.