A group of Erika Sasaki, Director of the Central Institute for Experimental Animals (also serving as a specially appointed professor at the Center for Experimental Animals, Keio University) and Hideyuki Okano, Professor of Keio University School of Medicine, is the first in the world to create a primate model of immunodeficiency by genome editing. Successful publication in the American scientific journal "Cell Stem Cell".By making it possible to conduct research using primates that are closer to humans than mice, it is expected to contribute to the elucidation of the causes of various diseases and the development of treatment methods.

 Until now, genetically modified mice have been used for analysis of gene function and elucidation of various disease onset mechanisms, but in order to develop treatments for human diseases, primate model animals closer to humans are important. Will be. In 2009, the research group has already succeeded in creating the world's first "transgenic (* 1) marmoset" using a common marmoset, which is a small and highly fertile primate.However, the "target gene knockout technology (technology that destroys a specific gene to prevent it from functioning)" that has been used to create human disease model mice has not been applicable to primates.

 In this study, we succeeded in creating an "immunodeficient marmoset," which has significantly fewer immune-related cells by modifying genes involved in immunity by artificially editing the genome of fertilized marmosets.No normal immune function was observed in this marmoset, and characteristics similar to those of severe congenital immunodeficiency in humans appeared.In addition, the immunodeficient marmosets can survive for more than a year in a hygienic, clean breeding room.

 In the future, this immunodeficiency marmoset is expected to be used as a disease model for human immunodeficiency in elucidating the pathophysiology and developing treatment methods, and transplanting human iPS cells will promote verification of the efficacy and safety of regenerative medicine. It is thought that it will be done.Furthermore, it has been suggested that the primate genome editing technology developed this time may be applicable to research on human psychiatric and neurological disorders such as autism and schizophrenia.

* 1) Transgenic
An organism that has become an individual by artificially introducing a specific foreign gene into a cell

Keio University

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