Development of disease monkey model for hereditary retinal disease by gene editing

2015 Fiscal Year Final Research Report

• Project/Area Number: 15K15641
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Ophthalmology
• Research Institution: 独立行政法人国立病院機構(東京医療センター臨床研究センター)
• Principal Investigator: Iwata Takeshi 独立行政法人国立病院機構(東京医療センター臨床研究センター), 分子細胞生物学研究部, 部長 (90374157)
• Co-Investigator(Kenkyū-buntansha): MIZOTA Atsushi 帝京大学, 医学部, 教授 (10239262) ; SHIMOZAWA Nobuhiro 医薬基盤研究所, 霊長類医科学研究センター, 主任研究員 (50300786)
• Project Period (FY): 2015-04-01 – 2016-03-31
• Keywords: 医歯薬学 / 外科系臨床医学 / 眼科学 / 眼生化学・分子生物学 / カニクイザル

Outline of Final Research Achievements

The primate eye has unique structural characteristic such as macula, which do not exist in experimental animals, mouse, rat, and zebrafish. In this study, CRISPR/Cas9 was used to specifically cut the genome DNA in the fertilized egg to develop gene manipulated cynomolgus macaque monkeys with hereditary retinal disease. The fertilized egg was injected with CRISPR/Cas9 vector to specifically cut gene responsible for inherited macula disease. ES cell were detected with DNA cleavage at the site targeted. The detection of targeted mutation in ES cells provides the possibility of new born mutation by continuation of further experiments. A single offspring was born recently born. The new methods of CRISPR/Cas9 will be used to create other inherited macula diseases.

Free Research Field

分子細胞生物学