Development of new gene-repair therapy for primary immunodeficiencies with artificial nuclease

2017 Fiscal Year Final Research Report

• Project/Area Number: 15K15393
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Pediatrics
• Research Institution: Fukuoka Children’s Hospital (2017); Kyushu University (2015-2016)
• Principal Investigator: HARA Toshiro 地方独立行政法人福岡市立病院機構福岡市立こども病院(臨床研究部), 臨床研究部, 院長 (40150445)
• Co-Investigator(Kenkyū-buntansha): 高田 英俊 九州大学, 医学研究院, 教授 (70294931) ; 山元 裕之 九州大学, 環境発達医学研究センター, 特任助教 (00710170)
• Project Period (FY): 2015-04-01 – 2018-03-31
• Keywords: 遺伝子治療 / 人工ヌクレアーゼ

Outline of Final Research Achievements

To examine the possibility of using gene therapy for primary immunodeficiencies, we constructed a helper-dependent adenovirus/adeno-associated virus hybrid vector to serve as a donor template, and a sequence-specific gRNA for double strand breaks. Human cord blood CD34 positive cells were infected with the hybrid vector and electroporated with gRNA and Cas9 protein. This delivery method can be a new gene-repair therapy for clinical application.

Free Research Field

小児科学