Gene editing trial could help find cure for sickle cell anemia

A treatment about to be tested in California holds the promise of possibly curing a debilitating disease, that disproportionately affects the African American community, sickle cell disease.

Researchers from UCSF, UC Berkeley, and UCLA are teaming up to learn if they can defeat the disease, by changing the patient's genetics.

For decades one of the best hopes for children or adults suffering from sickle cell has been a bone marrow transplant. Sometimes turning into a life or death struggle to find a match. But soon there could be a revolutionary alternative.

"That's challenging because you have to find a compatible donor. In this case, we're using the patient as their own donor," explains Dr. Mark Walters, M.D. of UCSF.

Dr. Walters is leading a groundbreaking clinical trial at UCSF Benioff Children's Hospital in Oakland First it helps to understand that sickle cell is a genetic condition that turns a patient's red blood cells into a hardened, sickle shape which can leave the blood unable to carry normal oxygen to the body.

"It's a hereditary disorder. It's caused by this one base pair change in their genome. And by correcting that mistake can we then cure the disease," he says.

To do that, they've turned to a Nobel prize-winning gene-editing technology developed by researcher Jennifer Doudna, Ph.D., and colleagues at U.C. Berkeley and the Innovative Genomics Institute. Known as CRISPR, the system allows doctors to identify genes that cause disease and then replace them with new DNA - a process known as gene editing.

In this case, the doctors plan to extract blood-producing stem cells from the patients, modify the genes, and place them back in their system to produce healthy red blood cells.

"Because they're healthy red blood cells they'll last longer in the circulation than the sickle red cells and over time they get enriched. So over time they'll take over all the blood capacity in the circulation," he adds.

Potentially curing the condition. And if they're successful, researchers believe the CRISPR strategy could eventually be used to treat a wide variety of genetic diseases.

"We're entering an age where we can seriously talk about, as Jennifer put it to me once, where CRISPR becomes the standard of medical care," says Fyodor Urnov, Ph.D., of U.C. Berkeley and the Innovative Genomics Institute.

The project received nearly seven million dollars in funding from the California Institute of Regenerative medicine. The trial is expected to launch this summer and will track six adults and three adolescents with severe sickle cell disease over four years. All in the hopes of re-writing the script for a dangerous and sometimes deadly disease.
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