2月23日，日本东京大学医学部准教授野入英世和该校研究生院教授中村荣一领导的联合研究小组公布最新研究成果称，他们成功地向动物体内植入了可运载基因的富勒烯（C60），以用于医学治疗。这是世界上以富勒烯为载体植入基因的首次报告，该研究成果拉开了开发低毒性、高功能基因植入法的序幕。相关论文发表于本周出版的美国《国家科学院院刊》上。
富勒烯是由60个碳原子结合成的球状分子，广泛应用于各种工业产品的制造。研究小组把普通的富勒烯合成为具有4个氨基的水溶性富勒烯，并将制造绿色荧光蛋白（GFP）的基因的DNA与水溶性富勒烯结合，注入到小鼠静脉中。随后，研究人员在小鼠的肺部、肝脏和脾脏中发现了绿色荧光蛋白基因，这证明绿色荧光蛋白被制造了出来。
目前的基因导入方法利用的是病毒和脂质类物质，但由于稳定性和安全性等各种问题无法克服，尚未达到实用阶段。脂质型基因试剂与DNA结合缺乏稳定性，利用静脉注射将基因输送到的脏器官仅限于肺部，而且在植入基因后，会导致肝脏和肾脏出现功能性障碍。相比之下，富勒烯基因试剂完全不会引发任何功能性障碍，非常安全。研究小组通过富勒烯输送对糖尿病有治疗效果的胰岛素基因的DNA，结果显示，导入动物体内的基因起作用后，血液中的胰岛素浓度上升，血糖下降。
水溶性富勒烯与DNA结合后成为100纳米左右的纳米粒子。这种纳米粒子在细胞内易于吸收，反之会再次还原成水溶性富勒烯和DNA。自日本科学家2000年发表了通过水溶性富勒烯和纳米管运载基因的研究以来，利用碳簇化合物成功向动物体内植入基因尚属世界首次。由于水溶性富勒烯具有低毒性，易于大量合成且成本低廉的优点，今后利用富勒烯的基因植入法将大有作为。
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《国家科学院院刊》发表论文摘要（英文）
Published online before print March 1, 2010, doi: 10.1073/pnas.0909223107
In vivo gene delivery by cationic tetraamino fullerene
Rui Maeda-Mamiyaa,b, Eisei Noirib,1, Hiroyuki Isobec, Waka Nakanishic, Koji Okamotob, Kent Doib, Takeshi Sugayad, Tetsuro Izumie, Tatsuya Hommaa, and Eiichi Nakamuraa,1
Abstract
Application of nanotechnology to medical biology has brought remarkable success. Water-soluble fullerenes are molecules with great potential for biological use because they can endow unique characteristics of amphipathic property and form a self-assembled structure by chemical modification. Effective gene delivery in vitro with tetra(piperazino)fullerene epoxide (TPFE) and its superiority to Lipofectin have been described in a previous report. For this study, we evaluated the efficacy of in vivo gene delivery by TPFE. Delivery of enhanced green fluorescent protein gene (EGFP) by TPFE on pregnant female ICR mice showed distinct organ selectivity compared with Lipofectin; moreover, higher gene expression by TPFE was found in liver and spleen, but not in the lung. No acute toxicity of TPFE was found for the liver and kidney, although Lipofectin significantly increased liver enzymes and blood urea nitrogen. In fetal tissues, neither TPFE nor Lipofectin induced EGFP gene expression. Delivery of insulin 2 gene to female C57/BL6 mice increased plasma insulin levels and reduced blood glucose concentrations, indicating the potential of TPFE-based gene delivery for clinical application. In conclusion, this study demonstrated effective gene delivery in vivo for the first time using a water-soluble fullerene.
