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Biokinetic studies of non-complexed siRNA versus nano-sized PEI F25-LMW/siRNA polyplexes following intratracheal instillation into mice
Publication date: 16 March 2016 Source:International Journal of Pharmaceutics, Volume 500, Issues 1–2 Author(s): Jens Lipka, Manuela Semmler-Behnke, Alexander Wenk, Jana Burkhardt, Achim Aigner, Wolfgang Kreyling Successful gene therapy requires stability and sufficient bioavailability of the applied drug at the site of action. In the case of RNA interference (RNAi), non-viral vectors play a promising role for delivering intact siRNA molecules. We selected a low molecular weight polyethyleneimine (PEI F25-LMW) and investigated the biokinetics of PEI F25-LMW/siRNA polyplexes in comparison to non-complexed siRNA mo...
Source: International Journal of Pharmaceutics - January 31, 2016 Category: Drugs & Pharmacology Source Type: research

Cholic acid-based mixed micelles as siRNA delivery agents for gene therapy
In this study, we demonstrate the use of lipids as co-surfactants for the preparation of mixed micelles to improve the siRNA delivery of cholic acid-based block copolymers. Poly(allyl glycidyl ether) (PAGE) and poly(ethylene glycol) (PEG) were polymerized on the surface of cholic acid to afford a star-shaped block copolymer with four arms (CA-PAGE-b-PEG)4. The allyl groups of PAGE were functionalized to bear primary or tertiary amines and folic acid was grafted onto the PEG chain end to increase cell uptake. (CA-PAGE-b-PEG)4 functionalized with either primary or tertiary amines show high siRNA complexation with close to 10...
Source: International Journal of Pharmaceutics - January 26, 2020 Category: Drugs & Pharmacology Source Type: research

Combination of photodynamic therapy and gene silencing achieved through the hierarchical self-assembly of porphyrin-siRNA complexes
Publication date: Available online 31 July 2019Source: International Journal of PharmaceuticsAuthor(s): Nabila Laroui, Maëva Coste, Laure Lichon, Yannick Bessin, Magali Gary-Bobo, Geneviève Pratviel, Colin Bonduelle, Nadir Bettache, Sébastien UlrichAbstractIn this work, we implemented a supramolecular approach in order to combine photodynamic therapy (PDT) with gene therapy. We made use of a simple cationic guanidylated porphyrin (H2‑PG) with the hypothesis that porphyrin aggregates should be capable of complexing siRNA through multivalent interactions and thus contribute to its intracellular delivery, while remaining...
Source: International Journal of Pharmaceutics - August 1, 2019 Category: Drugs & Pharmacology Source Type: research

Anticancer siRNA cocktails as a novel tool to treat cancer cells. Part (B). Efficiency of pharmacological action
Publication date: 15 May 2015 Source:International Journal of Pharmaceutics, Volume 485, Issues 1–2 Author(s): Volha Dzmitruk , Aleksandra Szulc , Dzmitry Shcharbin , Anna Janaszewska , Natallia Shcharbina , Joanna Lazniewska , Darya Novopashina , Marina Buyanova , Maksim Ionov , Barbara Klajnert-Maculewicz , Rafael Gómez-Ramirez , Serge Mignani , Jean-Pierre Majoral , Maria Angeles Muñoz-Fernández , Maria Bryszewska This paper examines a perspective to use newly engineered nanomaterials as effective and safe carriers for gene therapy of cancer. Three different groups of cationic dendrimers (PAMAM, phosphorus, and c...
Source: International Journal of Pharmaceutics - March 27, 2015 Category: Drugs & Pharmacology Source Type: research

Poly (amidoamine) (PAMAM) dendrimer mediated delivery of drug and pDNA/siRNA for cancer therapy
Publication date: 30 July 2018 Source:International Journal of Pharmaceutics, Volume 546, Issues 1–2 Author(s): Jun Li, Huamin Liang, Jing Liu, Ziyuan Wang Poly (amidoamine) (PAMAM) dendrimers are well-defined, highly branched macromolecules with numerous active amine groups on the surface. Because of their unique properties, PAMAM dendrimers have steadily grown in popularity in drug delivery, gene therapy, medical imaging and diagnostic application. This review focuses on the recent developments on the application in PAMAM dendrimers as effective carriers for drug and gene (pDNA, siRNA) delivery in cancer therapy, incl...
Source: International Journal of Pharmaceutics - May 26, 2018 Category: Drugs & Pharmacology Source Type: research

Carbosilane dendrimers with phosphonium terminal groups are low toxic non-viral transfection vectors for siRNA cell delivery
Publication date: Available online 13 March 2019Source: International Journal of PharmaceuticsAuthor(s): Regina Herma, Dominika Wrobel, Michaela Liegertová, Monika Müllerová, Tomáš Strašák, Marek Maly, Alena Semerádtová, Marcel Štofik, Dietmar Appelhans, Jan MalyAbstractNon-viral gene delivery vectors studied in the gene therapy applications are often designed with the cationic nitrogen containing groups necessary for binding and cell release of nucleic acids. Disadvantage is a relatively high toxicity which restricts the in vivo use of such nanoparticles. Here we show, that the 3rd generation carbosilane dendrim...
Source: International Journal of Pharmaceutics - March 13, 2019 Category: Drugs & Pharmacology Source Type: research

Chitosan as a non-viral co-transfection system in a cystic fibrosis cell line
This study shows proof-of-principle that co-transfection with chitosan might be an effective delivery system in a human CF cell line. It also offers a potential alternative to further develop therapeutic strategies for inherited disease treatments, such as CF. Graphical abstract
Source: International Journal of Pharmaceutics - February 22, 2016 Category: Drugs & Pharmacology Source Type: research

RNA-based Therapy for Osteogenesis
Publication date: Available online 5 August 2019Source: International Journal of PharmaceuticsAuthor(s): Pinpin Wang, Federico Perche, Delphine Logeart-Avramoglou, Chantal PichonAbstractNucleic acid-based therapy has shown great promise in accelerating bone regeneration as well as other diseases. Nucleic acids used in gene therapy mainly are either plasmid DNA (pDNA) or RNAs. Although pDNA therapy has been extensively studied for decades with encouraging preclinical and clinical results, side effects, and low efficiency associated with nuclear trafficking are hard to bypass. Unlike pDNA, RNAs (mRNA, siRNA, miRNA) exert the...
Source: International Journal of Pharmaceutics - August 6, 2019 Category: Drugs & Pharmacology Source Type: research

Ammonium salt modified mesoporous silica nanoparticles for dual intracellular-responsive gene delivery
Publication date: 25 September 2016 Source:International Journal of Pharmaceutics, Volume 511, Issue 2 Author(s): Yujie Li, Mingyang Hei, Yufang Xu, Xuhong Qian, Weiping Zhu Effective gene delivery system plays an importmant role in the gene therapy. Mesoporous silica nanoparticle (MSN) has become one potential gene delivery vector because of its high stability, good biodegradability and low cytotoxicity. Herein, MSN-based dual intracellular responsive gene delivery system CMSN-A was designed and fabricated. Short chain ammonium group, which is modified with disulfide bond and amide bond simultaneously, is facilely grafte...
Source: International Journal of Pharmaceutics - August 4, 2016 Category: Drugs & Pharmacology Source Type: research

Surfactant effect on the physicochemical characteristics of cationic solid lipid nanoparticles
In this study, three different cationic lipids (dioctadecyl dimethyl ammonium bromide, cetyltrimethyl ammonium bromide, cetylpyridinium chloride) and Brij 76 as nonionic surfactant were employed to formulate Precirol ATO 5 based cSLN using pEGFP-LUC as model plasmid. The physicochemical properties of cSLN were influenced by both type and amount of surfactants. Thermal analyses of bulk cSLN showed endothermic peaks significantly different from the ones of the single pure components, hinting a complete entanglement of the lipid matrix with the surfactants and justifying the different behavior of the cSLN in the ability to in...
Source: International Journal of Pharmaceutics - November 27, 2016 Category: Drugs & Pharmacology Source Type: research