An Engineered Complement Factor H Construct for Treatment of C3 Glomerulopathy.

ABSTRACT: 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, the serum t_1/2 of FH^{1-5^18-20} was significantly shorter than that of serum-purified FH.Methods We introduced the oligomerization domain of human FH-related protein 1 (denoted by R1-2) at the carboxy or amino terminus of h

SUBMITTER: Yang Y 

PROVIDER: S-EPMC6054357 | biostudies-literature | 2018 Jun

REPOSITORIES: biostudies-literature