Role of PIEZO1 in pathogenesis of sickle cell disease

  • Research type

    Research Study

  • Full title

    Role of PIEZO1 in pathogenesis of sickle cell disease

  • IRAS ID

    357804

  • Contact name

    Rasiqh Wadud

  • Contact email

    rw544@cam.ac.uk

  • Sponsor organisation

    University of Cambridge

  • Duration of Study in the UK

    3 years, 0 months, 0 days

  • Research summary

    This study will investigate why red blood cells in people with sickle cell disease let ions and water pass through too easily, and how we might correct this. In sickle cell disease, the usual oxygen-carrying protein (haemoglobin) is changed to a form called HbS. When oxygen levels fall, HbS can clump together and distort red cells into a stiff ‘sickle’ shape. At the same time the cells become leakier: they lose water and shrink, which raises the concentration of HbS and makes further sickling more likely. Calcium also enters the cells and triggers other changes, including a lipid molecule called phosphatidylserine (PS) moving to the cell surface that makes red cells stickier. Together, the shape change, dehydration and extra stickiness increase the chance of small blood vessels becoming blocked, which can cause pain crises, chest problems, stroke and organ damage.
    One membrane ‘gate’, called PIEZO1, seems central to these changes. It is a mechanosensitive channel which is activated by sickling and then lets in calcium with all the deleterious sequelae. The project, funded by the Wellcome Trust, will examine factors which activate or inhibit PIEZO1. The aim is to block PIEZO1 and thereby reduce the complications of SCD and support patient management.
    We will collect an anonymised small sample of fresh blood (5-10 ml) from patients attending sickle cell clinics. Thereafter, all work will be carried out in the laboratory and patients are not further involved except being fully informed of progress.

  • REC name

    London - Central Research Ethics Committee

  • REC reference

    25/PR/1467

  • Date of REC Opinion

    5 Dec 2025

  • REC opinion

    Further Information Favourable Opinion