Development of cynomolgus monkey with hereditary retinal diseases

2014 Fiscal Year Final Research Report

• Project/Area Number: 26670762
• 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): 2014-04-01 – 2015-03-31
• Keywords: 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

Number of hereditary retinal diseases in human is related to photoreceptors in the macula. The primate has unique structural and functional characteristic, which often becomes a problem using mouse or fish model of human retinal diseases. In this study, CRISPR/Cas9 was used to specifically cut the genome DNA in the fertilized egg to develop gene manipulated cynomolgus monkeys with hereditary retinal diseases. The fertilized egg was injected at the nucleus with CRISPR/Cas9 DNA vector and both in vitro culture and embryo transplantation was performed. ES cells were detected with DNA cleavage at the site targeted. However, one new born monkey derived from injected fertilized egg did not carry the mutation suggesting the germ line mutation was not achieved. The detection of targeted mutation in ES cells provides the possibility of new born mutation by continuation of further experiments. New methods of CRISPR/Cas9 will be tried to increase the efficiency of specific genome DNA cleavage.

Free Research Field

医歯薬学