What is it about?

Sickle cell disease is one of the most common genetic diseases in the US. Individuals with sickle cell disease suffer from painful vaso-occlusive episode (VOE), which often causes organ damage and dysfunction. While some therapies are approved to reduce its frequency, patients still suffer from VOE, and current treatments are primarily to relieve pain. Here, we discovered the importance of a multimeric protein, called von Willebrand factor (VWF), in the origin and development of VOE and documented a potential VWF-targeting treatment to reduce VOE-related organ damages.

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Why is it important?

In our study, VWF was found to be involved in the development of VOE. This conclusion is supported by the alleviation of anemia and VOE-related organ damage in a sickle cell disease mouse model in which VWF expression was deleted. ADAMTS13 is a protease that acts like a pair of scissors to cut VWF and reduce its activity. When ADAMTS13 was administrated, the sickle-cell-diseased mice appeared less sick: the mice had improved anemia and reduced vaso-occlusions in different organs.

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This page is a summary of: Endothelial VWF is critical for the pathogenesis of vaso-occlusive episode in a mouse model of sickle cell disease, Proceedings of the National Academy of Sciences, August 2022, Proceedings of the National Academy of Sciences,
DOI: 10.1073/pnas.2207592119.
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