Abstract
Gene therapy is an attractive treatment option for diseases of genetic origin, including several cancers and cardiovascular diseases. While viruses are effective vectors for delivering exogenous genes to cells, concerns related to insertional mutagenesis, immunogenicity, lack of tropism, decay and high production costs necessitate the discovery of non-viral methods. Significant efforts have been focused on cationic polymers as non-viral alternatives for gene delivery. Recent studies have employed combinatorial syntheses and parallel screening methods for enhancing the efficacy of gene delivery, biocompatibility of the delivery vehicle, and overcoming cellular level barriers as they relate to polymermediated transgene uptake, transport, transcription, and expression. This review summarizes and discusses recent advances in combinatorial syntheses and parallel screening of cationic polymer libraries for the discovery of efficient and safe gene delivery systems.
Keywords: Parallel screening, polymer library, transfection, gene delivery, transgene expression, cytotoxicity, gene therapy, complex biological barriers, physicochemical diversities, DNA binding efficacy, biodegradability
Combinatorial Chemistry & High Throughput Screening
Title: Discovery of Cationic Polymers for Non-Viral Gene Delivery Using Combinatorial Approaches
Volume: 14 Issue: 10
Author(s): Sutapa Barua, James Ramos, Thrimoorthy Potta, David Taylor, Huang-Chiao Huang, Gabriela Montanez and Kaushal Rege
Affiliation:
Keywords: Parallel screening, polymer library, transfection, gene delivery, transgene expression, cytotoxicity, gene therapy, complex biological barriers, physicochemical diversities, DNA binding efficacy, biodegradability
Abstract: Gene therapy is an attractive treatment option for diseases of genetic origin, including several cancers and cardiovascular diseases. While viruses are effective vectors for delivering exogenous genes to cells, concerns related to insertional mutagenesis, immunogenicity, lack of tropism, decay and high production costs necessitate the discovery of non-viral methods. Significant efforts have been focused on cationic polymers as non-viral alternatives for gene delivery. Recent studies have employed combinatorial syntheses and parallel screening methods for enhancing the efficacy of gene delivery, biocompatibility of the delivery vehicle, and overcoming cellular level barriers as they relate to polymermediated transgene uptake, transport, transcription, and expression. This review summarizes and discusses recent advances in combinatorial syntheses and parallel screening of cationic polymer libraries for the discovery of efficient and safe gene delivery systems.
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Cite this article as:
Barua Sutapa, Ramos James, Potta Thrimoorthy, Taylor David, Huang Huang-Chiao, Montanez Gabriela and Rege Kaushal, Discovery of Cationic Polymers for Non-Viral Gene Delivery Using Combinatorial Approaches, Combinatorial Chemistry & High Throughput Screening 2011; 14 (10) . https://dx.doi.org/10.2174/138620711797537076
DOI https://dx.doi.org/10.2174/138620711797537076 |
Print ISSN 1386-2073 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5402 |
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