CRISPR Gene Editing Having Amazing Results

Scientist replacing part of a DNA molecule

Jennifer Doudna was born in Washington, D.C. She grew up in Hilo, Hawaii. After graduating from Pomona College in 1985, she earned a Ph.D. from Harvard Medical School in 1989. Doudna is a renowned American biochemist known for her pioneering work in CRISPR gene editing. She was awarded the 2020 Nobel Prize in Chemistry. In addition to being a professor at UC Berkeley, she is also president and chair of the board of the Innovative Genomics Institute, a faculty scientist at Lawrence Berkeley National Laboratory, a senior investigator at the Gladstone Institutes, and an adjunct professor of cellular and molecular pharmacology at the University of California, San Francisco. It is brilliant and dedicated people like Jennifer Doudna who are creating breakthrough after breakthrough and saving lives.  

Doudna is the go-to figure for what is referred to as the “CRISPR revolution”. In 2012, Doudna and Emmanuelle Charpentier were the first to propose that CRISPR could be used for programmable editing of genomes. The technique is now considered one of the most significant discoveries in the history of biology.

I first wrote about CRISPR technology in 2016. It is not exactly a household name. CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. If you read the details about CRISPR, it might make your head hurt. It is really complex. CRISPR was discovered in 2007 and is partly based on what a yogurt company unearthed about a bacteria which could eliminate viruses. The technology has evolved dramatically over the last few years into a gene editing technique which may become a powerful tool for the cure of multiple diseases, including cancer. You will be seeing the term CRISPR more frequently in the months ahead.

The words cancer and cure will be used together more and more. Laboratory experiments have shown it is possible to literally eliminate cancer tumors. Today’s chemotherapy treatments are aimed at killing the cancer cells. Sometimes it works and sometimes it does not. The side effects can be severe. The gene editing approach is designed to modify our DNA and have our body fight the cancer instead of the chemo. I think of CRISPR technology like video editing. A video clip consists of a series of video frames. A video editor enables you to remove frames which might be irrelevant or unappealing. You then save the modified video.

Sickle cell disease (SCD) is a disease for which those afflicted may benefit greatly from CRISPR. SCD affects millions of people throughout the world, especially those whose ancestors came from sub-Saharan Africa; Spanish-speaking regions in the Western Hemisphere (South America, the Caribbean, and Central America); Saudi Arabia; India; and Mediterranean countries such as Turkey, Greece, and Italy.

Healthy red blood cells are round, and they move smoothly through our blood vessels to carry oxygen throughout the body. For those with SCD, the red blood cells become hard and sticky and look like a C-shaped farm implement, hence the name sickle. The sickle cells are short lived, and as a result the patient has a constant shortage of red blood cells. When sickle cells move through small blood vessels, they can get stuck and clog the blood flow causing pain and other serious and life-threatening effects.

A clinical trial with ten patients has shown some amazing results. The treatment is complex. Doctors remove stem cells from the patients’ bone marrow and use CRISPR to edit a gene in the cells. The result is the production of fetal hemoglobin, just like what is produced by fetuses in the womb. The patients are given chemotherapy to destroy most of their bone marrow. This is a grueling step but it is needed to make room for the gene-edited cells. The last step is the infusion of billions of new cells into the patient’s body.

Researchers reported this month the first 10 patients treated with the CRISPR gene-editing responded well, although enduring the intense chemotherapy step. Jennifer Doudna was quoted in NPR Health News, “I’m very excited to see these results. Patients appear to be cured of their disease, which is simply remarkable.”

The early stage of CRISPR was difficult and viewed as highly risky. A gene-editing trial with a volunteer teenager in 1999 ended in his tragic death. This was a setback of years, but much has been learned. The University of Pennsylvania, where the trial took place, became very cautious but is now advancing the technology. Other trials have included participants who have sarcoma, melanoma or myeloma. The end goal of the CRISPR technology is to create living drugs which can be put in our bodies to eradicate tumor cells. Progress is much more imminent than when I wrote the 2016 article. What I have read and heard from experts suggests it is not far away to say cancer has finally been eliminated.

See other stories I wrote about CRISPR here.

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