In 2019, Victoria Gray became the first person in the world to receive a CRISPR-based treatment for sickle cell disease. Seven years later, she has not experienced a sickle cell crisis since the one-time treatment — a remarkable outcome that illustrates how decades of basic scientific research can eventually transform the treatment of inherited diseases.Gray’s journey, and the science behind her treatment, are detailed in a January 2026 **Berkeley News** article by Anne Brice, published as part of the university’s *Berkeley Voices* series. The story traces Gray’s life with sickle cell disease and the development of CRISPR gene editing at the University of California, Berkeley.
Diagnosed at just three months old
Gray was only three months old when she began crying inconsolably at her home in Goodman, Mississippi. Doctors initially could not determine the cause, but blood tests eventually revealed that she had sickle cell disease.In 1985, when Gray was diagnosed, the outlook for children with the condition was far worse than it is today. She grew up knowing that her illness could shorten her life. As a child, she would listen to conversations between her mother and grandmother and understood that something was seriously wrong.“Unlike normal kids, for my birthday I would wish for another year of life,” Gray recalled in the Berkeley News account.Sickle cell disease is caused by an inherited genetic mutation that affects haemoglobin, the protein in red blood cells responsible for carrying oxygen. The condition can cause red blood cells to become rigid and crescent-shaped, blocking blood vessels and reducing oxygen delivery.For Gray, severe pain crises occurred repeatedly and often required hospitalisation, blood transfusions and pain medication.
The Berkeley science behind CRISPR
While Gray was growing up with the disease, researchers were working on a technology that would eventually offer a radically different approach to treating genetic disorders.UC Berkeley biochemist Jennifer Doudna and French microbiologist Emmanuelle Charpentier helped develop CRISPR-Cas9, a gene-editing system that allows scientists to make targeted changes to DNA.Their landmark research, published in 2012, helped establish CRISPR as a powerful tool for editing genetic material. UC Berkeley has described the technology as capable of targeting and modifying DNA in bacteria, plants, animals and human cells.The key question for sickle cell disease was how to use this technology without simply attempting to repair the original mutation.Researchers instead looked at fetal haemoglobin, a form of haemoglobin naturally produced before birth. Scientists discovered that a protein called BCL11A plays a major role in switching off fetal haemoglobin after birth.The idea was simple but potentially transformative: use gene editing to alter that switch and allow the body to produce fetal haemoglobin again.
Victoria Gray volunteered for an experimental treatment
In 2019, Gray was being evaluated in Nashville, Tennessee, for a bone marrow transplant when her doctor, Haydar Frangoul, offered her another option: participation in an experimental CRISPR therapy trial.She agreed to become the first person with sickle cell disease to receive the treatment.Doctors first collected millions of her stem cells. The cells were sent to a laboratory, where they were edited using CRISPR. Gray then underwent high-dose chemotherapy to destroy her existing bone marrow and make space for the edited cells.On July 2, 2019, the edited cells were infused back into her body.Doudna later described Gray’s decision as extraordinary.“Imagine being the first person who volunteered to be number one when something has never been tested before in humans,” Doudna said, according to Berkeley News. “It’s amazing to think of how brave she had to be.”
‘Is this what normal feels like?’
The treatment was far from easy. Gray lost her hair after chemotherapy, developed painful sores in her mouth and throat and spent weeks in hospital while her immune system recovered and the edited stem cells became established in her bone marrow.But the outcome changed her life.She gradually stopped needing blood transfusions and reduced her dependence on pain medication. Seven or eight months after the treatment, she woke up without pain.“I was like, ‘Oh, my God. This is what normal feels like?’” she recalled.Gray eventually stopped using a walker and was able to perform everyday activities independently. She also returned to work and, most importantly, became more involved in her children’s lives.“CRISPR not only freed me, it freed my children,” Gray said.
From one patient to an approved CRISPR therapy
Gray’s treatment was experimental, but the technology has since moved into mainstream medicine.In December 2023, the US Food and Drug Administration approved Casgevy, the first CRISPR-based therapy for sickle cell disease. The treatment was developed by Vertex Pharmaceuticals and CRISPR Therapeutics, building on the scientific foundation created by researchers including Doudna and her collaborators.The advance has also come with significant challenges. The US list price for Casgevy is more than $2 million, while patients may also face lengthy hospital stays, travel and other expenses associated with receiving the treatment.Doudna has argued that reducing the cost of gene-editing therapies will be essential if such treatments are to reach more patients.“We want to make sure that the technology continues to advance, and not just as a technology in academic papers, but in real-world applications,” she said.
Why CRISPR could change medicine
Sickle cell disease is only one area where researchers are exploring CRISPR. Scientists are investigating gene-editing approaches for cancer, cardiovascular disease and other inherited disorders.Doudna has also pointed to the potential of gene editing in tissues beyond blood and the liver as delivery technologies improve. In a 2025 Berkeley News talk, she highlighted Gray as an example of how CRISPR had already moved from laboratory research into a treatment that could dramatically change a patient’s life.For Gray, the scientific breakthrough has become a personal mission. She now works as a patient advocate and public speaker, sharing her experience with patients, medical professionals, researchers and organisations.“I think it’s important to share my story,” she said. “We’re real people with real lives.”Her story offers a powerful lesson in how science progresses: a discovery that begins as curiosity-driven research in a university laboratory can, years later, become a treatment capable of changing a patient’s life.
