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An extended mini-complement factor H molecule ameliorates experimental C3 glomerulopathy.

Kidney international (2015-07-30)
Eva-Maria Nichols, Thomas D Barbour, Isabel Y Pappworth, Edwin K S Wong, Jeremy M Palmer, Neil S Sheerin, Matthew C Pickering, Kevin J Marchbank
RESUMEN

Abnormal regulation of the complement alternative pathway is associated with C3 glomerulopathy. Complement factor H is the main plasma regulator of the alternative pathway and consists of 20 short consensus repeat (SCR) domains. Although recombinant full-length factor H represents a logical treatment for C3 glomerulopathy, its production has proved challenging. We and others have designed recombinant mini-factor H proteins in which 'non-essential' SCR domains have been removed. Here, we report the in vitro and in vivo effects of a mini-complement factor H protein, FH

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MISSION® esiRNA, targeting mouse C3