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U Researchers Helped Bring Novel Gene Therapy to Clinical Trials

Parent holds child's hand in hospital bed

University of Minnesota researchers’ work played a pivotal role in helping scientists launch the first-ever clinical trials that involve editing a gene inside the body to cure several rare genetic diseases.

A story from the Associated Press earlier this month announced that Sangamo Therapeutics, a biotechnology company based in Richmond, CA, has begun injecting clinical trial participants with gene editing therapies that aim to help people with Hurler syndrome (MPS I), Hunter syndrome (MPS II), and hemophilia B. U of M Medical School researchers Dr. Chester Whitley, Ph.D., professor of pediatrics, and Scott McIvor, Ph.D., professor of genetics, cell biology, and development, conducted sponsored research to help Sangamo complete preclinical studies in animal models and advance toward clinical trials.

U of M faculty and staff also assisted the company in designing the protocol for the clinical trials. Whitley is currently one of several principal investigators for the clinical trials, which are being conducted at sites throughout the US.

The genetic disorders, which first show symptoms early in childhood, stem from the body’s inability to produce enzymes it needs to function correctly. Unlike previous gene editing therapies—which involve removing cells from the body, modifying them, and returning them to the body—the clinical trial aims to edit cells from inside the body, “reprogramming” the DNA to produce the needed enzymes.

Kevin Coss

Kevin Coss

Kevin is a writer with the Office of the Vice President for Research.

coss@umn.edu

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