Regents of the University of Minnesota;Recombinetics, Inc.;Board of Regents of The University of Texas System
发明人:
Scott C. Fahrenkrug,Daniel F. Carlson,Peter Igarashi,Thomas Carroll
申请号：
US15771243
公开号：
US20190254266A1
申请日:
2016.10.27
申请国别(地区):
US
年份:
2019
代理人:
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 debilitate 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.
