Development of gene repair therapy using autologous cells for primary immunodeficiencies

2014 Fiscal Year Final Research Report

• Project/Area Number: 22249043
• Research Category: Grant-in-Aid for Scientific Research (A)
• Allocation Type: Single-year Grants
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Kyushu University
• Principal Investigator: HARA Toshiro 九州大学, 医学(系)研究科(研究院), 教授 (40150445)
• Co-Investigator(Kenkyū-buntansha): TAKADA Hidetoshi 九州大学, 医学研究院, 教授 (70294931) ; IHARA Kenji 大分大学, 医学部, 教授 (80294932) ; MIYAWAKI Toshio 富山大学, 医学系研究科, 教授 (10143885) ; NAKAHATA Tatsutoshi 京都大学, iPS細胞研究所, 教授 (20110744) ; NAKATSU Yoshimichi 九州大学, 医学研究院, 准教授 (00207820)
• Co-Investigator(Renkei-kenkyūsha): ISHIKAWA Fumihiko 理化学研究所, 免疫・アレルギー科学総合研究センター・ヒト疾患モデル研究グループ, グループディレクター (30403918)
• Project Period (FY): 2010-04-01 – 2015-03-31
• Keywords: 遺伝子修復治療

Outline of Final Research Achievements

We constructed a HD-Ad.AAV BTK targeting vector that is composed of the genomic sequence containing BTK exons 6-19, TIMM8A and GFP/hygromycin resistant gene. By using HD-Ad.AAV. BTK vector, we found that the targeting in the BTK gene occurred in 4 among 755 hygromycin-resistant BFU-E colonies differentiated from the cord blood CD34+ cells. Importantly, the gene targeting was also observed in CD19+ lymphoid progenitor cells that could be differentiated from the homologous recombinants of CD34+ cells. Our study shows the potential of HD-Ad.AAV. BTK vector for the BTK gene therapy by homologous recombination in hematopoietic stem cells.

Free Research Field

小児科学