An altering method that has the potential to treat hemophilia

A group of researchers from Italy and the United States worked with several companies to find that adding a protein to a lentiviral vector protects them from the immune system. In a paper published in the journal Science Translational Medicine, the team described their work in finding a new way to treat hemophilia and how their methods work in monkeys.

Hemophilia is an inherited bleeding disorder that slows the body's ability to form blood clots. When most people bleed, the process that is known in their bodies as the "coagulation cascade" usually stops bleeding. Platelets condense or accumulate at the wound, forming a clot. Then, the body's coagulation factors work together to form a more permanent blockage in the wound. Hemophilia patients lack or have low levels of clotting factors, so blood does not condense properly.

Hemophilia patients are prone to bleeding, and blood clotting takes a long time. The extent of hemophilia symptoms depends on the severity of clotting factor deficiency. People who are mildly deficient may bleed in the event of trauma, and people with severe defects may bleed for no reason.

According to the World Federation of Hemophilia (WFH), about one in every 10,000 people are born with this disease. There is currently no cure for hemophilia, but treatment can be used to prevent and treat bleeding episodes and to minimize complications associated with hemophilia.

Currently, scientists have been trying to use gene therapy to treat people with hemophilia. One such treatment is the use of adeno-associated virus (AAV) to deliver a gene that expresses a missing coagulation factor. Unfortunately, most adults are infected with the AAV virus, so they have an immune response to AAV. In this new study, the researchers used another approach using different types of vectors.

The researchers used Lentiviral Vectors (LVs) instead of AAV. LV is derived from HIV and is less common in humans, thus providing a better alternative to gene therapy. However, the researchers found that white blood cells are often cleared from the body by phagocytic cells (white blood cells) before they are delivered to target cells.

To solve this problem, the researchers used a protein called CD47. Previous studies have found that CD47 can be prevented from being detected and cleared by phagocytic cells. The researchers applied it to the surface of the LVs to help them escape detection and subsequent clearance.

The researchers injected the modified LVs into six monkeys and tested them. They observed that LVs were able to pass treatments to their targets (liver and spleen) and effectively transfer genes to liver cells as planned.

Although more tests are needed to verify the feasibility of this approach, the researchers noted that their method may reduce the therapeutic dose of hemophilia and may minimize side effects.

Cite this article

CUSABIO team. An altering method that has the potential to treat hemophilia.


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