CRISPR Center Advances Genetic Disease Research

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Elaine Zhang, a project manager at the Innovative Genomics Institute (IGI), conducts genetic research using CRISPR technology. (Photo courtesy of the IGI)

A formidable collaboration between three University of California (UC) schools and leading global life sciences and diagnostics innovator the Danaher Corporation heralds a new era in the fight against rare and deadly genetic diseases, such as sickle cell disease — which predominantly impacts Black and Hispanic populations in the U.S. — through the innovative use of CRISPR technology.

Spearheaded by the Innovative Genomics Institute (IGI), this joint effort brings together genetics researchers and clinician experts from UC San Francisco, UC Los Angeles, UC Berkeley, and other research institutions, to expedite the development of curative therapies for diseases that have previously lacked effective treatments.

The Danaher-IGI Beacon for CRISPR Cures center will leverage genome editing technology to research a wide range of genetic disorders. The center, which will be led out of the IGI headquarters at UC Berkeley, combines expertise in genetics research, clinical practice, and industry resources to accelerate the development and deployment of CRISPR-based treatments. The goal is to establish new standards for safety and efficacy while streamlining the path from preclinical research to clinical trials.

“The unique nature of CRISPR makes it ideal for developing and deploying a platform capability for CRISPR cures on demand,” said Fyodor Urnov, PhD, IGI’s Director of Technology and Translation, in a press release. “Danaher and the IGI are in a unique position to potentially create a first-of-its-kind CRISPR cures ‘cookbook’ that could be used by any team wishing to take on other diseases.”

The center’s initial focus will be on hemophagocytic lymphohistiocytosis (HLH) and Artemis-deficient severe combined immunodeficiency (ART-SCID), two conditions characterized by defects in a patient’s immune system. Traditional treatments for these disorders, such as bone marrow transplants, often fall short due to complications.

By targeting specific gene mutations associated with these diseases, researchers hope to develop therapies that address their underlying causes, improve outcomes, and enhance quality of life for those affected.

Using CRISPR, the IGI has already made incredible advancements in treating sickle cell disease through clinical trials at the Comprehensive Sickle Cell Center at UC San Francisco Benioff Children’s Hospital in Oakland — which was established to address racial biases in health care. In 2021, the center received $17 million in funding to advance the use of CRISPR in sickle cell research.

“This therapy has the potential to transform sickle cell disease care,” said Mark Walters, MD, a pediatric professor at UC San Francisco and principal investigator of the clinical trials. “If this is successfully applied in young patients, it has the potential to prevent irreversible complications of the disease.”

Since then, researchers have been testing the possibility of replacing the gene that causes sickle cell with a healthy one manufactured using a patient’s own stem cells. Early tests have been positive, indicating a potential cure for the disease.

“With CRISPR, we can speed up the development of improved therapies that can reach all the patients who need them,” said Jennifer Puck, MD, a faculty member at the Jeffrey Modell Diagnostic Center for Primary Immunodeficiencies and Institute for Human Genetics, both at UC San Francisco. “All patients deserve a sense of urgency.— including those with rare diseases, many of whom are children.”