Campus news

Born at UC Berkeley: a breakthrough in the treatment of sickle cell disease

Discover how CRISPR, a technology co-created by a UC Berkeley professor, is being used to transform medicine.

A sickle is a crescent-shaped blade once used to harvest wheat. When red blood cells take on that same curved shape, it signals sickle cell disease –– an inherited condition that causes cells to become stiff and sticky, blocking blood flow and triggering episodes of severe, stabbing pain known as vaso-occlusive crises.

Sickle cell disease affects more than 100,000 people in the United States, with an outsized impact on the Black community, and an estimated 8 million worldwide. It remains a significant cause of childhood mortality globally.

For decades, treatment options were limited to managing symptoms. That began to change with the development of CRISPR, a powerful gene-editing technology that traces its roots to the University of California, Berkeley. In this short video, part of our ongoing video series Born at UC Berkeley, learn about the innovation from our student host, Angela Zhang.

CRISPR, short for “clustered regularly interspaced short palindromic repeats,” allows scientists to precisely alter DNA. The technology was co-created by UC Berkeley professor Jennifer Doudna and French scientist Emmanuelle Charpentier, who were awarded the Nobel Prize in Chemistry in 2020 for the discovery. Doudna holds the Li Ka Shing Chancellor’s Chair in Biomedical and Health Sciences, and is a professor of biochemistry, biophysics and structural biology.  

“It’s a way of changing the code of life in a precise fashion,” as Doudna has explained in the past.

Sickle cell disease is caused by a single genetic mutation that produces an abnormal protein in red blood cells. CRISPR-based therapies target that underlying genetic error rather than just treating its effects.

In 2023, the Food and Drug Administration approved the first CRISPR-based treatment for sickle cell disease, known as Casgevy, marking a historic milestone in medicine. The therapy began reaching patients in specialized centers in 2024.

While gene-editing therapies hold enormous promise, they are costly and difficult to access. Researchers at Berkeley and elsewhere are now working to improve delivery methods and reduce barriers, with the goal of making these life-changing treatments available far beyond a handful of hospitals.