Loading patent…
AssigneeRegeneron Pharmaceuticals, Inc.
InventorsJeffery Haines, Keith Crosby, Meghan Drummond Samuelson, David Frendewey, Brian Zambrowicz, Andrew J. Murphy
Estimating the term…
Non-human animal cells and non-human animals comprising a humanized TTR locus comprising a beta-slip mutation and methods of using such non-human animal cells and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized TTR locus comprising a beta-slip mutation express a human transthyretin protein or a chimeric transthyretin protein, fragments of which are from human transthyretin. Methods are provided for using such non-human animals comprising a humanized TTR locus to assess in vivo efficacy of human-TTR-targeting reagents such as nuclease agents designed to target human TTR.
A non human animal mouse comprising in its genome a genetically modified endogenous Ttr locus, wherein a region of the endogenous Ttr locus comprising both Ttr coding sequence and non-coding sequence has been deleted and replaced with a corresponding human TTR sequence comprising both TTR coding sequence and non-coding sequence, and wherein the genetically modified endogenous Ttr locus comprises a mutation that causes a shift in beta-strand D of the encoded transthyretin protein,
A mouse cell comprising in its genome a genetically modified endogenous Ttr locus, wherein a region of the endogenous Ttr locus comprising both Ttr coding sequence and non-coding sequence has been deleted and replaced with a corresponding human TTR sequence comprising both TTR coding sequence and non-coding sequence, and wherein the genetically modified endogenous Ttr locus comprises a mutation that causes a shift in beta-strand D of the encoded transthyretin protein,
Dependencies are inferred from the claim text.