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Expanding the Therapeutic Potential of Platelets

Milwaukee — December 01, 2023
Written By Christian Kastrup, PhD

New research from Christian Kastrup, PhD, could lead to better treatments for excessive bleeding, cancer and sepsis.


Blood has several components that all play important roles in human health. Red blood cells carry oxygen throughout the body, white blood cells help to protect us from infection, plasma transports nutrients, and platelets help your blood to clot and stop bleeding.

Platelets have long been used to treat patients with severe bleeding, cancer, sepsis and more. Because of their multifunctional roles in a variety of diseases, researchers are interested in learning more about how they function and how to engineer them to create new cellular therapies for patients. But despite platelets’ potential, currently no effective methods exist for genetically modifying platelets to express specific therapeutic proteins.

Nucleic acids lie at the center of the issue. Nucleic acids are sort of the libraries of cells, housing the information that helps cells to become more intelligent. When cells read this information, they create RNA, which acts as a messenger and instructs cells on how they should function. However, researchers have had a difficult time introducing genetically modified nucleic acids to platelets in order to treat diseases.

New research from Versiti Blood Research Institute Senior Investigator Christian Kastrup, PhD, explores the use of lipid nanoparticles (LNPs), or tiny fat particles, to deliver messenger RNA (mRNA) to platelets in order to introduce the synthesis of new proteins to fight disease. In a recent paper in the journal Science Advances, Dr. Kastrup and his team describe an approach that uses platelet-optimized LNPs containing mRNA to enable this protein expression.

To accomplish this, they tested a library of mRNA-LNP and synthesized proteins that did not require, nor correlate with, platelet activation. They found that transfected platelets, or platelets that were introduced to the new proteins, were well-tolerated in in-vivo models of bleeding, retaining their normal functionality and accumulating to stop bleeding.

This knowledge and success with genetically engineered platelets will help researchers like Dr. Kastrup expand the therapeutic potential of platelets.

Read the full article in Science Advances.

About the expert: Christian Kastrup, PhD, is a senior investigator at Versiti Blood Research Institute and a professor in the Department of Surgery, Division of Trauma and Acute Care Surgery and the Departments of Biochemistry, Biomedical Engineering, and Pharmacology and Toxicology at the Medical College of Wisconsin.

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