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7.9
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Pharmaceutics
Special Issues
Gene Therapy
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Gene Therapy

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

Deadline for manuscript submissions: closed (31 July 2011) | Viewed by 54319

Special Issue Editors

Prof. Dr. Yvonne Perrie

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Guest Editor
Strathclyde Institute of Pharmacy and Biomedical Sciences, University of Strathclyde, Glasgow G4 0RE, UK
Interests: particulate systems and formulations for drug delivery; vaccine adjuvanticity and diagnostics
Special Issues, Collections and Topics in MDPI journals
Dr. Defang Ouyang

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Guest Editor
Aston Pharmacy School, School of Life and Health Sciences, Aston University, Aston Triangle, Birmingham B4 7ET, UK
Interests: design, physicochemical characterization and development of non-viral systems for gene delivery; stability of protein/peptide formulation using physicochemical methods and computer simulation

Keywords

  • gene therapy
  • gene delivery
  • siRNA
  • antisense therapy
  • viral vectors
  • non-viral vectors

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Published Papers (6 papers)

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Research

Jump to: Review

18 pages, 860 KiB  
Article
Hydrotalcite Intercalated siRNA: Computational Characterization of the Interlayer Environment
by Hong Zhang, Defang Ouyang, Vinuthaa Murthy, Yunyi Wong, Zhiping Xu and Sean C. Smith
Pharmaceutics 2012, 4(2), 296-313; https://doi.org/10.3390/pharmaceutics4020296 - 7 Jun 2012
Cited by 8 | Viewed by 8430
Abstract
Using molecular dynamics (MD) simulations, we explore the structural and dynamical properties of siRNA within the intercalated environment of a Mg:Al 2:1 Layered Double Hydroxide (LDH) nanoparticle. An ab initio force field (Condensed-phase Optimized Molecular Potentials for Atomistic Simulation Studies: COMPASS) [...] Read more.
Using molecular dynamics (MD) simulations, we explore the structural and dynamical properties of siRNA within the intercalated environment of a Mg:Al 2:1 Layered Double Hydroxide (LDH) nanoparticle. An ab initio force field (Condensed-phase Optimized Molecular Potentials for Atomistic Simulation Studies: COMPASS) is used for the MD simulations of the hybrid organic-inorganic systems. The structure, arrangement, mobility, close contacts and hydrogen bonds associated with the intercalated RNA are examined and contrasted with those of the isolated RNA. Computed powder X-ray diffraction patterns are also compared with related LDH-DNA experiments. As a method of probing whether the intercalated environment approximates the crystalline or rather the aqueous state, we explore the stability of the principle parameters (e.g., the major groove width) that differentiate both A- and A'- crystalline forms of siRNA and contrast this with recent findings for the same siRNA simulated in water. We find the crystalline forms remain structurally distinct when intercalated, whereas this is not the case in water. Implications for the stability of hybrid LDH-RNA systems are discussed. Full article
(This article belongs to the Special Issue Gene Therapy)
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17 pages, 1107 KiB  
Article
Exploring the Correlation Between Lipid Packaging in Lipoplexes and Their Transfection Efficacy
by Behfar Moghaddam, Sarah E. McNeil, Qinguo Zheng, Afzal R. Mohammed and Yvonne Perrie
Pharmaceutics 2011, 3(4), 848-864; https://doi.org/10.3390/pharmaceutics3040848 - 18 Nov 2011
Cited by 27 | Viewed by 10657
Abstract
Whilst there is a large body of evidence looking at the design of cationic liposomes as transfection agents, correlates of formulation to function remain elusive. In this research, we investigate if lipid packaging can give further insights into transfection efficacy. DNA lipoplexes composed [...] Read more.
Whilst there is a large body of evidence looking at the design of cationic liposomes as transfection agents, correlates of formulation to function remain elusive. In this research, we investigate if lipid packaging can give further insights into transfection efficacy. DNA lipoplexes composed of 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE) or 1,2-distearoyl-sn-glycero-3-phosphoethanolamine (DSPE) in combination with 1,2-dioleoyl-3-trimethylammonium-propane (DOTAP) or 1,2-stearoyl-3-trimethylammonium-propane (DSTAP) were prepared by the lipid hydration method. Each of the formulations was prepared by hydration in dH2O or phosphate buffer saline (PBS) to investigate the effect of buffer salts on lipoplex physicochemical characteristics and in vitro transfection. In addition, Langmuir monolayer studies were performed to investigate any possible correlation between lipid packaging and liposome attributes. Using PBS, rather than dH2O, to prepare the lipoplexes increased the size of vesicles in most of formulations and resulted in variation in transfection efficacies. However, one combination of lipids (DSPE:DOTAP) could not form liposomes in PBS, whilst the DSPE:DSTAP combination could not form liposomes in either aqueous media. Monolayer studies demonstrated saturated lipid combinations offered dramatically closer molecular packing compared to the other combinations which could suggest why this lipid combination could not form vesicles. Of the lipoplexes prepared, those formulated with DSTAP showed higher transfection efficacy, however, the effect of buffer on transfection efficiency was formulation dependent. Full article
(This article belongs to the Special Issue Gene Therapy)
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16 pages, 197 KiB  
Article
Fourth Generation Phosphorus-Containing Dendrimers: Prospective Drug and Gene Delivery Carrier
by D. Shcharbin, V. Dzmitruk, A. Shakhbazau, N. Goncharova, I. Seviaryn, S. Kosmacheva, M. Potapnev, E. Pedziwiatr-Werbicka, M. Bryszewska, M. Talabaev, A. Chernov, V. Kulchitsky, A.-M. Caminade and J.-P. Majoral
Pharmaceutics 2011, 3(3), 458-473; https://doi.org/10.3390/pharmaceutics3030458 - 5 Aug 2011
Cited by 52 | Viewed by 8777
Abstract
Research concerning new targeting delivery systems for pharmacologically active molecules and genetic material is of great importance. The aim of the present study was to investigate the potential of fourth generation (P4) cationic phosphorus-containing dendrimers to bind fluorescent probe 8-anilino-1-naphthalenesulfonate (ANS), anti-neoplastic drug [...] Read more.
Research concerning new targeting delivery systems for pharmacologically active molecules and genetic material is of great importance. The aim of the present study was to investigate the potential of fourth generation (P4) cationic phosphorus-containing dendrimers to bind fluorescent probe 8-anilino-1-naphthalenesulfonate (ANS), anti-neoplastic drug cisplatin, anti-HIV siRNA siP24 and its capability to deliver green fluorescent protein gene (pGFP) into cells. The interaction between P4 and ANS (as the model drug) was investigated. The binding constant and the number of binding centers per one molecule of P4 were determined. In addition, the dendriplex between P4 and anti-HIV siRNA siP24 was characterized using circular dichroism, fluorescence polarization and zeta-potential methods; the average hydrodynamic diameter of the dendriplex was calculated using zeta-size measurements. The efficiency of transfection of pGFP using P4 was determined in HEK293 cells and human mesenchymal stem cells, and the cytotoxicity of the P4-pGFP dendriplex was studied. Furthermore, enhancement of the toxic action of the anti-neoplastic drug cisplatin by P4 dendrimers was estimated. Based on the results, the fourth generation cationic phosphorus-containing dendrimers seem to be a good drug and gene delivery carrier candidate. Full article
(This article belongs to the Special Issue Gene Therapy)
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19 pages, 420 KiB  
Article
Self-Assembled Lipoplexes of Short Interfering RNA (siRNA) Using Spermine-Based Fatty Acid Amide Guanidines: Effect on Gene Silencing Efficiency
by Abdelkader A. Metwally and Ian S. Blagbrough
Pharmaceutics 2011, 3(3), 406-424; https://doi.org/10.3390/pharmaceutics3030406 - 13 Jul 2011
Cited by 11 | Viewed by 8223
Abstract
Four guanidine derivatives of N4,N9-diacylated spermine have been designed, synthesized, and characterized. These guanidine-containing cationic lipids bound siRNA and formed nanoparticles. Two cationic lipids with C18 unsaturated chains, N1,N12-diamidino-N4,N [...] Read more.
Four guanidine derivatives of N4,N9-diacylated spermine have been designed, synthesized, and characterized. These guanidine-containing cationic lipids bound siRNA and formed nanoparticles. Two cationic lipids with C18 unsaturated chains, N1,N12-diamidino-N4,N9-dioleoylspermine and N1,N12-diamidino-N4-linoleoyl-N9-oleoylspermine, were more efficient in terms of GFP expression reduction compared to the other cationic lipids with shorter C12 (12:0) and very long C22 (22:1) chains. N1,N12-Diamidino-N4-linoleoyl-N9-oleoylspermine siRNA lipoplexes resulted in GFP reduction (26%) in the presence of serum, and cell viability (64%). These data are comparable to those obtained with TransIT TKO. Thus, cationic lipid guanidines based on N4,N9-diacylated spermines are good candidates for non-viral delivery of siRNA to HeLa cells using self-assembled lipoplexes. Full article
(This article belongs to the Special Issue Gene Therapy)
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Review

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19 pages, 1131 KiB  
Review
Polyamidoamine Dendrimer Conjugates with Cyclodextrins as Novel Carriers for DNA, shRNA and siRNA
by Hidetoshi Arima, Keiichi Motoyama and Taishi Higashi
Pharmaceutics 2012, 4(1), 130-148; https://doi.org/10.3390/pharmaceutics4010130 - 1 Feb 2012
Cited by 38 | Viewed by 8631
Abstract
Gene, short hairpin RNA (shRNA) and small interfering RNA (siRNA) delivery can be particularly used for the treatment of diseases by the entry of genetic materials mammalian cells either to express new proteins or to suppress the expression of proteins, respectively. Polyamidoamine (PAMAM) [...] Read more.
Gene, short hairpin RNA (shRNA) and small interfering RNA (siRNA) delivery can be particularly used for the treatment of diseases by the entry of genetic materials mammalian cells either to express new proteins or to suppress the expression of proteins, respectively. Polyamidoamine (PAMAM) StarburstTM dendrimers are used as non-viral vectors (carriers) for gene, shRNA and siRNA delivery. Recently, multifunctional PAMAM dendrimers can be used for the wide range of biomedical applications including intracellular delivery of genes and nucleic acid drugs. In this context, this review paper provides the recent findings on PAMAM dendrimer conjugates with cyclodextrins (CyDs) for gene, shRNA and siRNA delivery. Full article
(This article belongs to the Special Issue Gene Therapy)
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16 pages, 2839 KiB  
Review
Adenovirus Vector-Derived VA-RNA-Mediated Innate Immune Responses
by Mitsuhiro Machitani, Tomoko Yamaguchi, Kahori Shimizu, Fuminori Sakurai, Kazufumi Katayama, Kenji Kawabata and Hiroyuki Mizuguchi
Pharmaceutics 2011, 3(3), 338-353; https://doi.org/10.3390/pharmaceutics3030338 - 11 Jul 2011
Cited by 33 | Viewed by 8492
Abstract
The major limitation of the clinical use of replication-incompetent adenovirus (Ad) vectors is the interference by innate immune responses, including induction of inflammatory cytokines and interferons (IFN), following in vivo application of Ad vectors. Ad vector-induced production of inflammatory cytokines and IFNs also [...] Read more.
The major limitation of the clinical use of replication-incompetent adenovirus (Ad) vectors is the interference by innate immune responses, including induction of inflammatory cytokines and interferons (IFN), following in vivo application of Ad vectors. Ad vector-induced production of inflammatory cytokines and IFNs also results in severe organ damage and efficient induction of acquired immune responses against Ad proteins and transgene products. Ad vector-induced innate immune responses are triggered by the recognition of Ad components by pattern recognition receptors (PRRs). In order to reduce the side effects by Ad vector-induced innate immune responses and to develop safer Ad vectors, it is crucial to clarify which PRRs and which Ad components are involved in Ad vector-induced innate immune responses. Our group previously demonstrated that myeloid differentiating factor 88 (MyD88) and toll-like receptor 9 (TLR9) play crucial roles in the Ad vector-induced inflammatory cytokine production in mouse bone marrow-derived dendritic cells. Furthermore, our group recently found that virus associated-RNAs (VA-RNAs), which are about 160 nucleotide-long non-coding small RNAs encoded in the Ad genome, are involved in IFN production through the IFN-β promoter stimulator-1 (IPS-1)-mediated signaling pathway following Ad vector transduction. The aim of this review is to highlight the Ad vector-induced innate immune responses following transduction, especially VA-RNA-mediated innate immune responses. Our findings on the mechanism of Ad vector-induced innate immune responses should make an important contribution to the development of safer Ad vectors, such as an Ad vector lacking expression of VA-RNAs. Full article
(This article belongs to the Special Issue Gene Therapy)
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