An engineered complement factor H construct for treatment of C3 glomerulopathy

Y Yang, H Denton, OR Davies… - Journal of the …, 2018 - journals.lww.com
Background C3 glomerulopathy (C3G) is associated with dysregulation of the alternative
pathway of complement activation, and treatment options for C3G remain limited.
Complement factor H (FH) is a potent regulator of the alternative pathway and might offer a
solution, but the mass and complexity of FH makes generation of full-length FH far from
trivial. We previously generated a mini-FH construct, with FH short consensus repeats 1–5
linked to repeats 18–20 (FH 1–5^ 18–20), that was effective in experimental C3G. However …