Complement system in diseases

The complement system is a network of proteins in the blood that forms one of the body's first lines of defense, tagging pathogens for destruction and triggering inflammation within seconds of sensing danger. When its regulation breaks down, the same machinery that protects healthy tissue can attack the body's own cells, driving conditions such as atypical hemolytic uremic syndrome, a rare but life-threatening disorder in which uncontrolled complement activation damages small blood vessels and destroys red blood cells. Research has clarified how genetic variants in regulatory proteins—alongside factors like ADAMTS13 deficiency—determine who is vulnerable, and the development of Eculizumab, a monoclonal antibody that blocks a key complement protein, has transformed treatment for some of these disorders. Open questions center on why complement dysregulation manifests so differently across patients with similar mutations, and on how to design therapies that suppress harmful activation without compromising the immune surveillance the system was built to provide.

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Keywords

ComplementImmuneThrombotic MicroangiopathiesHemolytic Uremic SyndromeEculizumabInnate Immunity