Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use
US11891618B2 · kind B2 · utility
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28Claims
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Key dates
| Filing date | Jun 3, 2020 |
| Grant date | Feb 6, 2024 |
| Priority date | — |
| Expiry date | Feb 19, 2042 |
Classification
- Technology area (CPC A)Human Necessities
- CPC primaryA01K2227/105
- WIPO fieldBiotechnology
- WIPO sectorChemistry
Abstract
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.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.