Groundbreaking Research from the Versiti Blood Research Institute
New Research from the Versiti Blood Research Institute Improves Treatments for Excessive Bleeding, Cancer, and Sepsis
New research from Versiti Blood Research Institute Senior Investigator Christian Kastrup, PhD, has been featured in a recent paper in the journal Science Advances.
In “Genetically Engineered Transfusable Platelets using mRNA-Lipid Nanoparticles," Dr. Kastrup 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 the paper, Dr. Kastrup and his team describe an approach that uses platelet-optimized LNPs containing mRNA to enable this protein expression.
This knowledge and success with genetically engineered platelets will help researchers like Dr. Kastrup expand the therapeutic potential of platelets.
“This technology, the first to genetically modify transfusable platelets, has the potential to create more effective platelet therapies, from targeting bleeding in hematological disorders or crises to the large range of diseases that platelets help us heal from,” said Dr. Kastrup.
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 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.
To accomplish this, Dr. Kastrup and his team 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.
To learn more, read the full article in Science Advances.
Located in Wauwatosa, Wisconsin, on the Milwaukee Regional Center campus, the Versiti Blood Research Institute's work is dedicated to hematology in all its facets, spanning basic, translational, and clinical research, all with one goal: to make people's lives better. To learn more about the Versiti Blood Research Institute, visit Versiti.org.
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Versiti, a national leader in blood health innovation, was formed with the mission to improve the health of patients and enable the success of our healthcare partners nationally. We provide innovative, value-added solutions in the fields of transfusion medicine, transplantation, and blood-related diseases to meet the needs of each of our customers. The collective efforts across Versiti result in improved patient outcomes, expanded access to care, and cost efficiencies for healthcare systems nationwide. For more information, visit versiti.org.