Nobel Prize winner and UC Berkeley professor Jennifer Doudna presented developments on her gene research at a UC Santa Barbara Arts & Lectures event on Oct. 22. Her most recent research on DNA sequence editing has been applied to treating sickle cell anemia and cancer. 

Dr. Jennifer Doudna presented her research on CRISPR-Cas9 treatments at The Granada Theater. Courtesy of UCSB

Doudna, who spoke at The Granada Theatre in downtown Santa Barbara, invented Clustered Regularly Interspaced Short Palindromic (CRISPR)-Cas9 technology in 2012, which eventually earned her a Nobel Prize in chemistry in 2020 for its applications to treating chronic diseases like cancer and sickle cell anemia. 

The newly Food and Drug Administration (FDA)-approved treatment for sickle cell anemia is the “first clinical therapy of CRISPR-Cas9,” Doudna said.

As a professor of chemistry, biochemistry and molecular biology at the University of California, Berkeley, Doudna leads the Doudna Lab, which employs undergraduate and graduate students to study the structure and mechanism of CRISPR-Cas9, as well as its application in diagnostics and gene editing.  

According to Harvard Medical School, 250 people worldwide have received CRISPR-Cas9 treatment so far. Doudna said that CRISPR-Cas9 gene editing has changed the way that labs around the world approach biomedical research. This includes UCSB’s own molecular, cellular and developmental biology department, with assistant professor Chris Richardson leading a lab investigating natural DNA repair in order to further improve gene editing tools like CRISPR-Cas9.

CRISPR-Cas9 employs naturally-occurring CRISPR DNA sequences, along with the Cas9 protein, to develop a system for cutting out pieces of DNA and changing the sequence entirely.

Doudna explained the origin of the CRISPR-Cas9 technology, which happened after she met Jillian Banfield, a microbiologist looking at the adaptive immune systems of bacteria through the mechanism of CRISPR. CRISPR provides a place where the bacterial cell can store a little piece of viral DNA, allowing the cell to detect a virus with the same sequence signature as the collected DNA and destroy it using its associated Cas9 protein. “We realized that we could harness the chemistry of this process,” said Doudna.

The most notable use of the technology on humans is in combating sickle cell anemia by maintaining the production of fetal hemoglobin, a protein that naturally ceases production with aging. This protein, according to Doudna, has a remarkable ability to treat the painful and sometimes fatal disease. 

The FDA approved the treatment in December 2023 as the first clinical therapy of CRISPR-Cas9. Doudna hopes to decrease costs and duration of treatment in the coming years, making the therapy more widely accessible. 

Doudna and her lab have since worked on targeting human T-cells, which help fight cancer in vivo. “In vivo” means that the editing may take place within the human body as opposed to “in vitro,” which happens in a petri dish. T-cells edited with CRISPR-Cas9 become chimeric antigen receptor T-cells and are thus strengthened in their ability to recognize cells on the surface of tumors and kill the tumor. 

The use of CRISPR-Cas9, Doudna said, comes at extreme risk due to its potential application on human stem cells. Editing stem cells creates alterations that are heritable, meaning they can be passed on to the organism’s offspring and can have widespread effects on the entire human genome. Doudna strongly opposes this use of the technology and promotes consistent guidelines and transparency through all uses of the CRISPR-Cas9 technology. 

Doudna also discussed the ability of CRISPR-Cas9 to combat climate change. Doudna said that reprogramming CRISPR-Cas9 to target cattles’ microbiomes could significantly reduce methane emissions on a wide scale. Cattle are the world’s main producers of methane, a greenhouse gas that propels global warming. 

Confident in the success of CRISPR-Cas9 gene editing, Doudna is hopeful that, with responsible use and increased FDA support, the technology will continue to revolutionize the way scientists approach diseases in humans and in the environment.

A version of this article appeared on p. 6 of the Oct. 31, 2024 edition of the Daily Nexus.