Special Issue "Delivery and Genomics of Large Molecules"

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A special issue of Pharmaceutics (ISSN 1999-4923).

Deadline for manuscript submissions: closed (31 August 2012)

Special Issue Editors

Guest Editor
Prof. Dr. David A Jans

Department of Biochemistry and Molecular Biology, Monash University, Clayton, Vic. 3800, Australia
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Guest Editor
Dr. Kylie Wagstaff

Nuclear Signalling Laboratory, Department of Biochemistry and Molecular Biology, Monash University, Clayton, Vic. 3800, Australia
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Special Issue Information

Dear Colleagues,

Both the study and treatment of numerous diseases require the delivery of large molecules to cells/tissues in a specific/controlled fashion. For example, during gene therapy applications, large pieces of DNA encoding multiple therapeutic genes (ranging in size from small plasmids to mini-chormosomes) must be delivered to the nuclei of specific cells to treat inherited or acquired disorders. Numerous complications arise from attempting to deliver such large macromolelcules meaning that considerations such as packaging, including the ability to coat and if necessary uncoat in controlled fashion, as well as delivery through numerous cellular and tissue specific barriers must be taken into account in the design of such systems. For instance, in the gene therapy example one must not only take into account the design and regulation of the genes encoded in the therapeutic vector, but also examine how the DNA is going to be condensed and protected from degradation and how it will traverse across the plasma membrane, through the crowded cytoplasm and across the nuclear envelope before being transcribed in the nucleus in regulated fashion. These challenges and many more define the field of large molecule delivery and will be the mainstay of work in this area for many years.

Prof. Dr. David A Jans
Dr. Kylie Wagstaff
Guest Editors

Related Journal

  • Genes - an Open Access journal of genetics and genomics.

Keywords

  • gene delivery
  • drug delivery
  • gene therapy
  • phenomics
  • nuclear transport
  • gene transfer
  • intracellular transport

Published Papers (1 paper)

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Research

Open AccessArticle Efficient Gene Silencing by Self-Assembled Complexes of siRNA and Symmetrical Fatty Acid Amides of Spermine
Pharmaceutics 2011, 3(2), 125-140; doi:10.3390/pharmaceutics3020125
Received: 28 January 2011 / Revised: 11 March 2011 / Accepted: 22 March 2011 / Published: 25 March 2011
Cited by 6 | PDF Full-text (401 KB) | HTML Full-text | XML Full-text
Abstract
Gene silencing by siRNA (synthetic dsRNA of 21-25 nucleotides) is a well established biological tool in gene expression studies and has a promising therapeutic potential for difficult-to-treat diseases. Five fatty acids of various chain length and oxidation state (C12:0, C18:0, C18:1, C18:2, C22:1)
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Gene silencing by siRNA (synthetic dsRNA of 21-25 nucleotides) is a well established biological tool in gene expression studies and has a promising therapeutic potential for difficult-to-treat diseases. Five fatty acids of various chain length and oxidation state (C12:0, C18:0, C18:1, C18:2, C22:1) were conjugated to the naturally occurring polyamine, spermine, and evaluated for siRNA delivery and gene knock-down. siRNA delivery could not be related directly to gene silencing efficiency as N4,N9-dierucoyl spermine resulted in higher siRNA delivery compared to N4,N9-dioleoyl spermine. GFP silencing in HeLa cells showed that the unsaturated fatty acid amides are more efficient than saturated fatty acid amides, with N4,N9-dioleoyl spermine resulting in the most efficient gene silencing in the presence of serum. The alamarBlue cell viability assay showed that fatty acid amides of spermine have good viability (75%–85% compared to control) except N4,N9-dilauroyl spermine which resulted in low cell viability. These results prove that unsaturated fatty acid amides of spermine are efficient, non-toxic, non-viral vectors for siRNA mediated gene silencing. Full article
(This article belongs to the Special Issue Delivery and Genomics of Large Molecules)
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