Abstract:
:Gene augmentation has been the paradigm in the majority of gene therapy protocols but in recent years the potential of repairing the mutated gene in situ by targeted gene correction has become a reality. In fact, targeted gene repair has many advantages over conventional replacement strategies, notably the possibility to treat dominant as well as recessive disorders, and the small molecular size of the pharmacologically active agent. Chimeric RNA/DNA oligonucleotides, small fragment homologous replacement, as well as triplex-forming and single-stranded oligonucleotides are all examples of the growing armamentarium for gene repair, and are the subject of this review. In addition, we have also included a discussion of the reawakened Sleeping Beauty (SB) transposon system as a novel non-viral gene replacement strategy.
journal_name
J Drug Targetjournal_title
Journal of drug targetingauthors
Richardson P,Thoma C,Kren BT,Steer CJdoi
10.1080/10611860290016739keywords:
subject
Has Abstractpub_date
2002-03-01 00:00:00pages
133-41issue
2eissn
1061-186Xissn
1029-2330journal_volume
10pub_type
杂志文章,评审abstract::I125 radiolabelled HSA (HSA-I125) was utilised as a probe to quantify protein adsorption onto polystyrene (PS) and Poloxamine 908 coated PS (PS-908) particles. Upon ageing of the HSA-I125 a dramatic increase in the amount of protein adsorbed onto the two particle systems was observed. This phenomenon was not due to la...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/10611869708995849
更新日期:1997-01-01 00:00:00
abstract::The biodistribution of orally and intravenously administered archaeosomes in mice was compared to that of archaeosomes containing either coenzyme Q10 (archaeosome-CoQ10), polyethylene glycol (archaeosome-PEG), or PEG plus CoQ10 (archaeosome-PEG-CoQ10). The archaeosome formulations were prepared by a reverse-phase evap...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/10611869909085521
更新日期:2000-01-01 00:00:00
abstract::An in vivo study has been performed in order to determine the influence of amphotericin B (AMB) molecular organization on the toxicity and activity of this drug in the treatment of experimental visceral leishmaniasis. Three formulations with similar composition but different drug molecular self-association in aqueous ...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/10611860400006596
更新日期:2004-01-01 00:00:00
abstract::Liposomes have been prepared from dipalmitoylphosphatidylcholine (DPPC), cholesterol (Chol) and dimethyldioctadecylammonium bromide (DDAB). The cationic vesicles adsorb to biofilms of the skin-associated bacteria Staphylococcus epidermidis, which have a negative charge. Encapsulation of the antibacterial drug vancomyc...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/10611869609015975
更新日期:1996-01-01 00:00:00
abstract::A simple method for the separation of polyalkylcyanoacrylate nanoparticles was developed using polyisohexylcyanoacrylate (PIHCA) as a model. Fluorescein isothiocyanate dextran 70 was used to label the nanoparticles. Ultracentrifugation onto a performed sucrose gradient allowed the easy elimination of the dextran and o...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/10611869509059216
更新日期:1995-01-01 00:00:00
abstract::A new drug-targeting system for CD13(+) tumors has been developed, based on ultrasound-sensitive nanobubbles (NBs) and cell-permeable peptides (CPPs). Here, the CPP-doxorubicin conjugate (CPP-DOX) was entrapped in the asparagine-glycine-arginine (NGR) peptide modified NB (CPP-DOX/NGR-NB) and the penetration of CPP-DOX...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/1061186X.2015.1058802
更新日期:2016-01-01 00:00:00
abstract::Acyclovir is an antiviral agent and it has been particularly used for the treatment of herpes simplex infections. The treatment of infection in the oral cavity is often difficult, because of insufficient drug concentration in saliva when acyclovir is administered via the oral route in conventional tablet form for syst...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/10611860600566548
更新日期:2006-01-01 00:00:00
abstract::Doxorubicin (DXR) was encapsulated in long-circulating liposomes, composed of ganglioside GM1 (GM1)/distearoylphosphatidylcholine (DSPC)/cholesterol (CH) (0.13:1:1 in molar ratio) and sized to approximately 100 nm in mean diameter, with 98% entrapping efficiency by the transmembrane pH gradient method. Free DXR, DXR-D...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/10611869308996086
更新日期:1993-01-01 00:00:00
abstract::Nanoparticles have shown great promise in the treatment of cancer, with a demonstrated potential in targeted drug delivery. Among a myriad of nanocarriers that have been recently developed, dendrimers have attracted a great deal of scientific interests due to their unique chemical and structural properties that allow ...
journal_title:Journal of drug targeting
pub_type: 杂志文章,评审
doi:10.3109/1061186X.2015.1052077
更新日期:2015-01-01 00:00:00
abstract::Biphenylylacetic acid (BPAA) was linked to the free hydroxyl group of 2,6-di-O-methyl-beta-Cyclodextrin (DM-beta-CyD) through an ester linkage to obtain the site specific release of the drug to the colon. The conjugate at 1:1 mole ratio was separated from the reaction mixture by semipreparative reverse-phase HPLC and ...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/10611860310001615965
更新日期:2003-05-01 00:00:00
abstract::Gene delivery systems face the problem not only of the route toward the cell and tissues in question, but also of the molecularly crowded environment of both the cytoplasm and the nucleus itself. One of the physical barriers in the cytoplasm for diffusing nanoparticles is an actin network. Here, we describe the findin...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/1061186X.2010.521159
更新日期:2010-12-01 00:00:00
abstract::The ultimate goal of bacterial based cancer therapy is to achieve non-toxic penetration and colonisation of the tumour microenvironment. To overcome this efficacy-limiting toxicity of anticancer immunotherapy, we have tested a therapy comprised of systemic delivery of a vascular disrupting agent to induce intratumoral...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/1061186X.2020.1850739
更新日期:2020-12-21 00:00:00
abstract::A biophysical model was developed, using Ascaris suum as a model gastrointestinal nematode, to provide quantitative perspectives into the microenvironmental pH within the water-filled, porous, negatively charged cuticle matrix of gastrointestinal nematodes. The central features of the model include (a) the constant ra...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/10611869409015888
更新日期:1994-01-01 00:00:00
abstract::A higher encapsulation rate was obtained using the dehydration-rehydration method compared with the reverse-phase evaporation technique in negative multilamellar vesicles with amikacin (AMK) (45% versus 15%; P < 0.05) and teicoplanin (TCP) (34% versus 25%; P < 0.05). The addition of 250 mM sucrose to AMK- or TCP-conta...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/10611869308996076
更新日期:1993-01-01 00:00:00
abstract::This study was aimed to generate a new agent, norcantharidin (NCTD) encapsulated liposomes modified with a novel murine anti-human CD19 monoclonal antibody 2E8 (2E8–NCTD–liposomes), to specifically target the B-lineage leukemia stem cells (B-LSCs) and their progeny in vitro. Our results have shown that the positive pe...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/10611861003649720
更新日期:2010-11-01 00:00:00
abstract::Oleanolic acid (OA) is a pentacyclic triterpenoid found in various plant species. Triterpenoid compounds have been shown to inhibit tumor proliferation and to induce apoptosis in cancer cells. We synthesized an OA derivative and evaluated its inhibitory effects on cell proliferation in human colon cancer. Radioisotope...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/1061186X.2013.851684
更新日期:2014-10-07 00:00:00
abstract::Drug systems targeting tumor cells using basic fibroblast growth factor (bFGF) have been widely reported. In this study, the peptide KRTGQYKLC (bFGFp), containing cysteine at the carboxyl termination of the bFGF-derived peptide, was applied as a novel ligand targeting tumor cells. bFGFp was conjugated with bovine seru...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/10611860600849498
更新日期:2006-09-01 00:00:00
abstract::The use of liposomes in the treatment of severe infections is under investigation. Classical liposomes which localize in cells of the mononuclear phagocyte system (MPS) can be exploited in two ways. First for targeting of macrophage modulators such as muramyl peptides or IFN-gamma, to stimulate the cells of the MPS to...
journal_title:Journal of drug targeting
pub_type: 杂志文章,评审
doi:10.3109/10611869408996811
更新日期:1994-01-01 00:00:00
abstract::The present study evaluates the prophylactic efficacy of α-tocopherol (α-TOH), resveratrol (RES), and coenzyme Q10 (CoQ10) co-loaded self-nanoemulsifying drug delivery system (α-TOH-RES-CoQ10 SNEDDS) in 7,12-Dimethylbenz[a]anthracene (DMBA) induced breast cancer model. SNEDDS formulation components were rationally sel...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/1061186X.2017.1298603
更新日期:2017-07-01 00:00:00
abstract::The aim of this study was to evaluate the pharmacokinetics (PK), tissue distribution, and the specific drug targeting of cefpiramide sodium-loaded liposomes (CPMS-Lips) compared with cefpiramide sodium solution (CPMS-Sol) in mice. CPMS-Lips were prepared by reverse phase evaporation method. In the PK and biodistributi...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/10611861003667607
更新日期:2011-01-01 00:00:00
abstract::An aqueous dispersion of self-organized phospholipid tubules has been utilized as the template for silica-deposited nanotubules (approximately 0.5 microm thick and >10 microm long) by a sol-gel method. The formation of the hybrid tubules was mechanistically investigated by controlled sol-gel reaction. The incorporatio...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/10611860802295581
更新日期:2008-11-01 00:00:00
abstract::Toxoplasmic encephalitis (TE) is the most common clinical manifestation of reactivated infection with Toxoplasma gondii in immunocompromised patients that is lethal if untreated. The combination of pyrimethamine plus sulfadiazine or clindamycin is the standard therapy for the treatment of TE, but these combinations ar...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/10611861003733995
更新日期:2011-02-01 00:00:00
abstract::HEK cells were transfected with the GFP gene using various vectors: naked DNA, lipofectamine, and both PLGA and P(FASA) plasmid-loaded nanospheres. All methods were assessed alone and with the use of chloroquine, a lysosomal enzyme inhibitor. Transfection efficiencies were determined and compared at various times post...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/1061186021000038373
更新日期:2002-09-01 00:00:00
abstract::The aim of the present study was to develop a polymeric delivery system for water-insoluble drug oridonin. Amphiphilic block copolymers, poly(epsilon-caprolactone)-poly(ethylene glycol)-poly (epsilon-caprolactone) (PCL-PEO-PCL), were synthesized by ring-opening polymerization of caprolactone initiated by the hydroxyl ...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/10611860802200938
更新日期:2008-07-01 00:00:00
abstract::Drug efflux by the membrane transporter P-glycoprotein (P-gp) plays a key role in multidrug resistance (MDR). In order to bypass P-gp, thus overcoming MDR, a hybrid peptide comprising a cell penetrating peptide (Tat) and a drug binding motif (DBM) has been developed to noncovalently bind and deliver doxorubicin (Dox) ...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.3109/10611860903548347
更新日期:2010-07-01 00:00:00
abstract::Based on the organotropic characteristics of bile acids towards the liver and the intestine, two novel compounds of the Bamet family, containing at least one bile acid moiety bound to platinum(II), have been synthesized and their cytostatic effect compared to their ability to become accumulated in tumor cells of hepat...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/1061186021000001841
更新日期:2002-08-01 00:00:00
abstract::Tumour-associated macrophages (TAMs) represent as much as 50% of the solid mass in different types of human solid tumours including lung, breast, ovarian and pancreatic adenocarcinomas. The tumour microenvironment (TME) plays an important role in the polarisation of macrophages into the M1 phenotype, which is tumour-s...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/1061186X.2019.1648478
更新日期:2020-02-01 00:00:00
abstract::A polymeric gene carrier was developed to deliver vascular endothelial growth factor (VEGF) small interfering RNA (siRNA) for prostate cancer cells in a target-specific manner. Prostate cancer-binding peptide (PCP) was conjugated with polyethylenimine (PEI) via a poly(ethylene glycol) (PEG) linker (PEI-PEG-PCP). The P...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/10611860902767232
更新日期:2009-05-01 00:00:00
abstract::Most therapeutic agents are delivered orally. Consequently, the major classes of therapeutically useful chemicals are partially lipophilic, small molecular weight compounds. They have reasonable permeability coefficient values across cell membranes, including those of intestinal epithelia and vascular endothelia. In c...
journal_title:Journal of drug targeting
pub_type: 杂志文章,评审
doi:10.1080/10611860410001693715
更新日期:2004-02-01 00:00:00
abstract::A promising strategy to carry genetic material to brain cells either in vitro or in vivo is using the LDL receptor (LDLr) on blood-brain barrier. LDLr naturally help to low density lipoproteins (LDL(S)) transporting across the BBB by endocytosis. Here we present the idea of using the LDLr-mediated pathway for transpor...
journal_title:Journal of drug targeting
pub_type: 杂志文章
doi:10.1080/10611860601148908
更新日期:2007-04-01 00:00:00