- 专利标题: COMPOSITIONS AND METHODS FOR CHIMERIC EMBRYO-ASSISTED ORGAN PRODUCTION
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申请号: EP16860830.5申请日: 2016-10-27
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公开(公告)号: EP3367785A2公开(公告)日: 2018-09-05
- 发明人: ASAKURA, Atsushi , ARAVALLI, Raj , CHEERAN, Maxin , DUTTON, James , GARRY, Daniel, J. , GARRY, Mary, G. , GRAHAM, Melanie , LI, Ling , LOW, Walter , PANOSKALTSIS-MORTARI, Angela , O'BRIEN, Timothy , STEER, Clifford , TOLAR, Jakup , CARLSON, Daniel, F. , FAHRENKRUG, Scott, C. , KOYANO-NAKAGAWA, Naoko , PARR, Ann
- 申请人: Regents of the University of Minnesota , Recombinetics, Inc.
- 申请人地址: 600 McNamara Alumni Center 200 Oak Street SE Minneapolis, MN 55455 US
- 专利权人: Regents of the University of Minnesota,Recombinetics, Inc.
- 当前专利权人: Regents of the University of Minnesota,Recombinetics, Inc.
- 当前专利权人地址: 600 McNamara Alumni Center 200 Oak Street SE Minneapolis, MN 55455 US
- 代理机构: Reitstötter Kinzebach
- 优先权: US201562246926P 20151027; US201562246927P 20151027; US201562246929P 20151027; US201562246947P 20151027; US201562247092P 20151027; US201562247096P 20151027; US201562247115P 20151027; US201562247117P 20151027; US201562247118P 20151027; US201562247122P 20151027
- 国际公布: WO2017075276 20170504
- 主分类号: A01K67/00
- IPC分类号: A01K67/00 ; C12N5/07 ; C12N15/85 ; A01N1/02 ; A61D19/04
摘要:
Human or humanized tissues and organs suitable for transplant are disclosed herein. Gene editing of a host animal provides a niche for complementation of the missing genetic information by donor stem cells. Editing of a host genome to knock out or disrupt genes responsible for the growth and/or differentiation of a target organ and injecting that animal at an embryo stage with donor stem cells to complement the missing genetic information for the growth and development of the organ. The result is a chimeric animal in which the complemented tissue (human/humanized organ) matches the genotype and phenotype of the donor. Such organs may be made in a single generation and the stem cell may be taken or generated from the patient's own body. As disclosed herein, it is possible to do so by simultaneously editing multiple genes in a cell or embryo creating a "niche" for the complemented tissue. Multiple genes can be targeted for editing using targeted nucleases and homology directed repair (HDR) templates in vertebrate cells or embryos.
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