Ultrapure chitosan oligomers as carriers for corneal gene transfer.

作者: Eytan A. Klausner , Zhong Zhang , Robert L. Chapman , Richard F. Multack , Michael V. Volin

DOI: 10.1016/J.BIOMATERIALS.2009.10.031

关键词:

摘要: NOVAFECT chitosans are ultrapure chitosan oligomers that were recently marketed as carriers for non-viral gene therapy. There no reports on systematic design and improvement of formulations based delivery. Therefore, we have designed characterized chitosan-DNA nanoparticles NOVAFECT. We found the size oligomeric is small, or=44.1+/-3.5 millivolt. In vitro transfection studies demonstrated ability to effectively transfect COS-7 cells. rat corneas, injection a select formulation into stroma showed (a) luciferase expression was 5.4 times greater than following administration polyethylenimine-DNA nanoparticles; (b) cells express transgene, green fluorescent protein, keratocytes (corneal fibroblasts). This study lays foundation evaluating pharmaceuticals corneal therapy, promising approach treatment acquired inherited diseases otherwise lead blindness. Oligomeric can also be evaluated ocular outside cornea, various additional therapy applications.

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