Complement factor H gene knockout rat as a model of C3 glomerulopathy
AssigneeRegeneron Pharmaceuticals, Inc.
InventorsKishor Devalaraja-Narashimha, Jeffrey D. Lee, Lori Morton
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Abstract
Rat cells and rats comprising an inactivated Cfh locus and methods of making and using such rat cells and rats are provided. The rats comprising an inactivated Cfh locus model C3 glomerulopathy (C3G). Methods are provided for using such rats comprising an inactivated Cfh locus to assess in vivo efficacy of putative C3G therapeutic agents.
Claims28
A genetically modified rat with a modification consisting of an inactivated endogenous Cfh locus, wherein the genetically modified rat is homozygous for the inactivated endogenous Cfh locus, wherein the genetically modified rat has one or more symptoms of C3 glomerulopathy (C3G),
- wherein the genetically modified rat has increased blood urea nitrogen levels at an age of between about 7 weeks and about 17 weeks compared to a wild type rat at an age of between about 7 weeks and about 17 weeks,
- wherein the genetically modified rat has a decreased lifespan compared to a wild type rat, and
- wherein the median lifespan of the genetically modified rat is less than 150 days.
Dependencies are inferred from the claim text.