演題詳細

ポスター / Poster

ポスター 47 (Poster 47) :細胞療法

print

日程
2013年10月11日(金)
時間
16:50 - 17:50
会場
ポスター会場 / Poster (ロイトン札幌 3F エメラルドABCD)
座長・司会
三浦 康生 (Yasuo Miura):1
1:京都大学医学部附属病院 輸血細胞治療部
 
前へ戻る

Establishment of novel cell-based therapy for hemophilia A

演題番号 : PS-1-361

松井 英人 (Hideto Matsui):1、杉本 充彦 (Mitsuhiko Sugimoto):1、辰巳 公平 (Kohei Tatsumi):2、大橋 一夫 (Kazuo Ohashi):2、嶋 緑倫 (Midori Shima):3、岡野 光夫 (Teruo Okano):2

1:Regulatory Medicine for Thrombosis, Nara Medical University, Kashihara, Japan、2:Institute of Advanced Biomedical Engineering and Science, Tokyo Women's Medical University, Tokyo, Japan、3:Pediatrics, Nara Medical University, Kashihara, Japan

 

Gene and cell therapy for hemophilia A is an attractive strategy because Factor VIII (FVIII) is a secreted protein and the tight regulation of its expression is not necessary. Moreover, continuous expression of FVIII levels as low as 1-5% of normal have been shown to substantially ameliorate the bleeding phenotype as well as quality of life in severe hemophilia A patients. Cell therapy for hemophilia A using endothelial progenitors has emerged as a treatment option. To evaluate the alternative approach with subcutaneous cell transplantation, canine FVIII-transduced blood outgrowth endothelial cells (BOECs) were cultured on temperature-responsive culture dishes to develop BOECs sheet that can enhance the cell viability without traditional bioabsorbable scaffold matrices. Subcutaneous cell sheet transplantation studies indicated that all hemophilia A mice that were treated with BOECs sheet without any prior treatment developed anti-canine FVIII antibodies. In contrast, between 2-11% plasma levels of canine FVIII were confirmed to sustain for up to 300 days without developing anti-canine FVIII antibodies when hemophilia A mice were treated with cyclophosphamide. Histological examination revealed that the transplanted BOECs sheets were structured as flat clusters without any cells infiltration. In conclusion, tissue engineering approach using genetically modified BOECs sheet is viable for persistent tissue survival and providing therapeutic values. This novel ex vivo gene transfer strategy can provide the safe and efficacious delivery of FVIII in hemophilia A.

前へ戻る