While gene therapy has seemed always just on the verge of being right around the corner, the limitation has always been delivery of the gene. How do you get the new gene to the right cells and activated? An in-vivo mice study in PNAS may take us closer to a usable delivery system. Rui Maeda-Mamiya of the University of Tokyo and others were able to get diabetic mice to increase their insulin levels after delivery of a insulin 2 gene by a water-soluble fullerene.
From the study abstract:
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.
PNAS Article Abstract: In vivo gene delivery by cationic tetraamino fullerene
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