Literature DB >> 18841447

Design and synthesis of N4,N9-disubstituted spermines for non-viral siRNA delivery--structure-activity relationship studies of siFection efficiency versus toxicity.

Moustafa K Soltan1, Hassan M Ghonaim, Mohamed El Sadek, M Abou Kull, Lubna Abd El-aziz, Ian S Blagbrough.   

Abstract

PURPOSE: To study the effect of sequentially changing the chain length, oxidation level, and charge distribution in N4,N9-diacyl and N4,N9-dialkyl spermines on siRNA formulation, and then to compare their lipoplex transfection efficiency in cell lines.
METHODS: Eight N4,N9-diacyl polyamines: N4,N9-[didecanoyl, dilauroyl, dimyristoyl, dimyristoleoyl, dipalmitoyl, distearoyl, dioleoyl and diretinoyl]-1,12-diamino-4,9-diazadodecane were synthesized. Their abilities to bind to siRNA and form nanoparticles were studied using a RiboGreen intercalation assay and particle sizing. Two diamides were also reduced to afford tetraamines N4,N9-distearyl- and N4,N9-dioleyl-1,12-diamino-4,9-diazadodecane. Delivery of fluorescein-labelled Label IT RNAi Delivery Control was studied in FEK4 primary skin cells and in an immortalized cancer cell line (HtTA), and compared with TransIT-TKO.
RESULTS: The design, synthesis, and structure-activity relationship studies of a series of N4,N9-disubstituted spermines as efficient vectors for non-viral siRNA delivery to primary skin and cancer cell lines is reported. These non-liposomal cationic lipids are promising siRNA carriers based on the naturally occurring polyamine spermine showing that C-18 is a better chain length as shorter chains are more toxic.
CONCLUSIONS: N4,N9-Distearoyl spermine and N4,N9-dioleoyl spermine are efficient siRNA formulation and delivery vectors, even in the presence of serum, comparable to TransIT-TKO. However, four positive charges distributed as in spermine was significantly more toxic.

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Year:  2008        PMID: 18841447     DOI: 10.1007/s11095-008-9731-z

Source DB:  PubMed          Journal:  Pharm Res        ISSN: 0724-8741            Impact factor:   4.200


  44 in total

1.  Tight control of gene expression in mammalian cells by tetracycline-responsive promoters.

Authors:  M Gossen; H Bujard
Journal:  Proc Natl Acad Sci U S A       Date:  1992-06-15       Impact factor: 11.205

2.  Novel spermine-based cationic gemini surfactants for gene delivery.

Authors:  G Ronsin; C Perrin; P Guédat; A Kremer; P Camilleri; A J Kirby
Journal:  Chem Commun (Camb)       Date:  2001-11-07       Impact factor: 6.222

3.  Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans.

Authors:  A Fire; S Xu; M K Montgomery; S A Kostas; S E Driver; C C Mello
Journal:  Nature       Date:  1998-02-19       Impact factor: 49.962

4.  Efficient in-vitro transfer of a 60-Mb mammalian artificial chromosome into murine and hamster cells using cationic lipids and dendrimers.

Authors:  G de Jong; A Telenius; S Vanderbyl; A Meitz; J Drayer
Journal:  Chromosome Res       Date:  2001       Impact factor: 5.239

5.  Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids.

Authors:  James Heyes; Lorne Palmer; Kaz Bremner; Ian MacLachlan
Journal:  J Control Release       Date:  2005-10-03       Impact factor: 9.776

6.  Rapid and sensitive ethidium bromide fluorescence quenching assay of polyamine conjugate-DNA interactions for the analysis of lipoplex formation in gene therapy.

Authors:  A J Geall; I S Blagbrough
Journal:  J Pharm Biomed Anal       Date:  2000-06       Impact factor: 3.935

7.  Plasmid DNA size does not affect the physicochemical properties of lipoplexes but modulates gene transfer efficiency.

Authors:  P Kreiss; B Cameron; R Rangara; P Mailhe; O Aguerre-Charriol; M Airiau; D Scherman; J Crouzet; B Pitard
Journal:  Nucleic Acids Res       Date:  1999-10-01       Impact factor: 16.971

8.  A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosis.

Authors:  F E Ruiz; J P Clancy; M A Perricone; Z Bebok; J S Hong; S H Cheng; D P Meeker; K R Young; R A Schoumacher; M R Weatherly; L Wing; J E Morris; L Sindel; M Rosenberg; F W van Ginkel; J R McGhee; D Kelly; R K Lyrene; E J Sorscher
Journal:  Hum Gene Ther       Date:  2001-05-01       Impact factor: 5.695

9.  A novel non-viral vector for DNA delivery based on low molecular weight, branched polyethylenimine: effect of molecular weight on transfection efficiency and cytotoxicity.

Authors:  D Fischer; T Bieber; Y Li; H P Elsässer; T Kissel
Journal:  Pharm Res       Date:  1999-08       Impact factor: 4.200

10.  Systemic administration of LPD prepared with CpG oligonucleotides inhibits the growth of established pulmonary metastases by stimulating innate and acquired antitumor immune responses.

Authors:  M M Whitmore; S Li; L Falo; L Huang
Journal:  Cancer Immunol Immunother       Date:  2001-12       Impact factor: 6.968

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  8 in total

1.  Bisethylnorspermine lipopolyamine as potential delivery vector for combination drug/gene anticancer therapies.

Authors:  Yanmei Dong; Jing Li; Chao Wu; David Oupický
Journal:  Pharm Res       Date:  2010-06-25       Impact factor: 4.200

2.  N1,N12-Diacyl spermines: SAR studies on non-viral lipopolyamine vectors for plasmid DNA and siRNA formulation.

Authors:  Hassan M Ghonaim; Shi Li; Ian S Blagbrough
Journal:  Pharm Res       Date:  2009-10-30       Impact factor: 4.200

3.  Quantitative silencing of EGFP reporter gene by self-assembled siRNA lipoplexes of LinOS and cholesterol.

Authors:  Abdelkader A Metwally; Ian S Blagbrough; Judith M Mantell
Journal:  Mol Pharm       Date:  2012-10-25       Impact factor: 4.939

4.  Efficient Gene Silencing by Self-Assembled Complexes of siRNA and Symmetrical Fatty Acid Amides of Spermine.

Authors:  Abdelkader A Metwally; Charareh Pourzand; Ian S Blagbrough
Journal:  Pharmaceutics       Date:  2011-03-25       Impact factor: 6.321

5.  Self-Assembled Lipoplexes of Short Interfering RNA (siRNA) Using Spermine-Based Fatty Acid Amide Guanidines: Effect on Gene Silencing Efficiency.

Authors:  Abdelkader A Metwally; Ian S Blagbrough
Journal:  Pharmaceutics       Date:  2011-07-13       Impact factor: 6.321

Review 6.  Lipophilic Polyamines as Promising Components of Liposomal Gene Delivery Systems.

Authors:  Pavel A Puchkov; Michael A Maslov
Journal:  Pharmaceutics       Date:  2021-06-21       Impact factor: 6.321

7.  Influence of oligospermines architecture on their suitability for siRNA delivery.

Authors:  Maha Elsayed; Vincent Corrand; Vidula Kolhatkar; Yuran Xie; Na Hyung Kim; Rohit Kolhatkar; Olivia M Merkel
Journal:  Biomacromolecules       Date:  2014-03-04       Impact factor: 6.988

8.  Allele-specific RNA interference rescues the long-QT syndrome phenotype in human-induced pluripotency stem cell cardiomyocytes.

Authors:  Elena Matsa; James E Dixon; Christopher Medway; Orestis Georgiou; Minal J Patel; Kevin Morgan; Paul J Kemp; Andrew Staniforth; Ian Mellor; Chris Denning
Journal:  Eur Heart J       Date:  2013-03-06       Impact factor: 29.983

  8 in total

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