Abstract
A number of human diseases stem from defective genes. One approach to treating such diseases is to replace, or override, the defective genes with normal genes, an approach called 'gene therapy'. However, the introduction of correctly functioning DNA into cells is a non-trivial matter, and cells must be coaxed to internalize, and then use, the DNA in the desired manner. A number of polymer-based synthetic systems, or 'vectors', have been developed to entice cells to use exogenous DNA. These systems work across the nano, micro and macro length scales, and have been under continuous development for two decades, with varying degrees of success. The design criteria for the construction of more-effective delivery vectors at each length scale are continually evolving. This review focuses on the most recent developments in polymer-based vector design at each length scale.
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Acknowledgements
The author thanks the many investigators who willingly shared their peer-reviewed and preliminary research results for inclusion in this review, and Anne Doody for her critical analysis of the manuscript. Financial support from the Whitaker Foundation, the Walter H. Coulter Foundation and the New York State Center for Advanced Technology is gratefully appreciated.
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Putnam, D. Polymers for gene delivery across length scales. Nature Mater 5, 439–451 (2006). https://doi.org/10.1038/nmat1645
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DOI: https://doi.org/10.1038/nmat1645
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