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Ultrapure chitosan oligomers as carriers for corneal gene transfer.

Title: Ultrapure chitosan oligomers as carriers for corneal gene transfer.
Authors: Klausner EA; Department of Pharmaceutical Sciences, Midwestern University Chicago College of Pharmacy, Downers Grove, IL 60515, USA. eklausner@midwestern.edu; Zhang Z; Chapman RL; Multack RF; Volin MV
Source: Biomaterials [Biomaterials] 2010 Mar; Vol. 31 (7), pp. 1814-20. Date of Electronic Publication: 2009 Oct 30.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Language: English
Journal Info: Publisher: Elsevier Science Country of Publication: Netherlands NLM ID: 8100316 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1878-5905 (Electronic) Linking ISSN: 01429612 NLM ISO Abbreviation: Biomaterials Subsets: MEDLINE
Imprint Name(s): Publication: : Amsterdam : Elsevier Science; Original Publication: [Guilford, England] : IPC Science and Technology Press, 1980-
MeSH Terms: Gene Transfer Techniques*; Chitosan/*chemistry ; Cornea/*metabolism ; Drug Carriers/*chemistry; DNA/chemistry ; Green Fluorescent Proteins/metabolism ; Luciferases/genetics ; Luciferases/metabolism ; Nanoparticles/chemistry ; Animals ; COS Cells ; Cells, Cultured ; Chlorocebus aethiops ; Electrophoretic Mobility Shift Assay ; Gene Expression Regulation ; Microscopy, Fluorescence ; Particle Size ; Rats ; Transfection
Abstract: NOVAFECT chitosans are ultrapure chitosan oligomers that were recently marketed as carriers for non-viral gene therapy. There are no reports on systematic design and improvement of formulations based on NOVAFECT chitosans for gene delivery. Therefore, we have designed and characterized chitosan-DNA nanoparticles based on NOVAFECT. We found that the size of oligomeric chitosan-DNA nanoparticles is small, or=44.1+/-3.5 millivolt. In vitro transfection studies demonstrated the ability of oligomeric chitosan-DNA nanoparticles to effectively transfect COS-7 cells. In rat corneas, injection of a select formulation of oligomeric chitosan-DNA nanoparticles into the stroma showed that (a) luciferase gene expression was 5.4 times greater than following administration of polyethylenimine-DNA nanoparticles; and (b) the cells that express the transgene, green fluorescent protein, were keratocytes (corneal fibroblasts). This study lays the foundation for evaluating oligomeric chitosan-DNA nanoparticles as pharmaceuticals for corneal gene therapy, a promising approach for the treatment of acquired and inherited corneal diseases that otherwise lead to blindness. Oligomeric chitosan-DNA nanoparticles can also be evaluated for the treatment of ocular diseases outside of the cornea, and for various additional gene therapy applications.; ((c) 2009 Elsevier Ltd. All rights reserved.)
Substance Nomenclature: 0 (Drug Carriers); 147336-22-9 (Green Fluorescent Proteins); 9007-49-2 (DNA); 9012-76-4 (Chitosan); EC 1.13.12.- (Luciferases)
Entry Date(s): Date Created: 20091103 Date Completed: 20100408 Latest Revision: 20191210
Update Code: 20260130
DOI: 10.1016/j.biomaterials.2009.10.031
PMID: 19879644
Database: MEDLINE

Journal Article; Research Support, Non-U.S. Gov't