Strategies for hepatic gene correction.

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 Target

authors

Richardson P,Thoma C,Kren BT,Steer CJ

doi

10.1080/10611860290016739

keywords:

subject

Has Abstract

pub_date

2002-03-01 00:00:00

pages

133-41

issue

2

eissn

1061-186X

issn

1029-2330

journal_volume

10

pub_type

杂志文章,评审
  • Human serum albumin as a probe for surface conditioning--a study of the ageing effect.

    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

    authors: Armstrong TI,Illum L

    更新日期:1997-01-01 00:00:00

  • Influence of coenzyme Q10 on tissue distribution of archaeosomes, and pegylated archaeosomes, administered to mice by oral and intravenous routes.

    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

    authors: Omri A,Makabi-Panzu B,Agnew BJ,Sprott GD,Patel GB

    更新日期:2000-01-01 00:00:00

  • Amphotericin B molecular organization as an essential factor to improve activity/toxicity ratio in the treatment of visceral leishmaniasis.

    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

    authors: Sánchez-Brunete JA,Dea MA,Rama S,Bolás F,Alunda JM,Torrado-Santiago S,Torrado JJ

    更新日期:2004-01-01 00:00:00

  • Encapsulation of vancomycin and gentamicin within cationic liposomes for inhibition of growth of Staphylococcus epidermidis.

    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

    authors: Sanderson NM,Jones MN

    更新日期:1996-01-01 00:00:00

  • A new method to isolate polyalkylcyanoacrylate nanoparticle preparations.

    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

    authors: Pinto-Alphandary H,Balland O,Couvreur P

    更新日期:1995-01-01 00:00:00

  • Cell-penetrating peptide-doxorubicin conjugate loaded NGR-modified nanobubbles for ultrasound triggered drug delivery.

    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

    authors: Lin W,Xie X,Deng J,Liu H,Chen Y,Fu X,Liu H,Yang Y

    更新日期:2016-01-01 00:00:00

  • A sustained release dosage form of acyclovir for buccal application: an experimental study in dogs.

    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

    authors: Değim T,Eğlen B,Ocak O

    更新日期:2006-01-01 00:00:00

  • Enhanced tumor targeting of doxorubicin by ganglioside GM1-bearing long-circulating liposomes.

    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

    authors: Unezaki S,Maruyama K,Ishida O,Takahashi N,Iwatsuru M

    更新日期:1993-01-01 00:00:00

  • Tweaking dendrimers and dendritic nanoparticles for controlled nano-bio interactions: potential nanocarriers for improved cancer targeting.

    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

    authors: Bugno J,Hsu HJ,Hong S

    更新日期:2015-01-01 00:00:00

  • Synthesis, characterization and in vitro evaluation of dimethyl-beta-cyclodextrin-4-biphenylylacetic acid conjugate.

    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

    authors: Ventura CA,Paolino D,Pedotti S,Pistarà V,Corsaro A,Puglisi G

    更新日期:2003-05-01 00:00:00

  • Biphasic interactions between a cationic dendrimer and actin.

    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

    authors: Ruenraroengsak P,Florence AT

    更新日期:2010-12-01 00:00:00

  • Virulence-attenuated Salmonella engineered to secrete immunomodulators reduce tumour growth and increase survival in an autochthonous mouse model of breast cancer.

    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

    authors: Augustin LB,Milbauer L,Hastings SE,Leonard AS,Saltzman DA,Schottel JL

    更新日期:2020-12-21 00:00:00

  • Biophysical model of the transcuticular excretion of organic acids, cuticle pH and buffer capacity in gastrointestinal nematodes.

    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

    authors: Sims SM,Ho NF,Magas LT,Geary TG,Barsuhn CL,Thompson DP

    更新日期:1994-01-01 00:00:00

  • Efficient entrapment of amikacin and teicoplanin in liposomes.

    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

    authors: Ravaoarinoro M,Toma E,Agbaba O,Morisset R

    更新日期:1993-01-01 00:00:00

  • Targeting of the B-lineage leukemia stem cells and their progeny with norcantharidin encapsulated liposomes modified with a novel CD19 monoclonal antibody 2E8 in vitro.

    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

    authors: Zhang J,Tang Y,Li S,Liao C,Guo X

    更新日期:2010-11-01 00:00:00

  • Characterization of oleanolic acid derivative for colon cancer targeting with positron emission tomography.

    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

    authors: Kim SM,Jeong IH,Yim MS,Chae MK,Kim HN,Kim DK,Kang CM,Choe YS,Lee C,Ryu EK

    更新日期:2014-10-07 00:00:00

  • Basic fibroblast growth factor-binding peptide as a novel targeting ligand of drug carrier to tumor cells.

    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

    authors: Terada T,Mizobata M,Kawakami S,Yabe Y,Yamashita F,Hashida M

    更新日期:2006-09-01 00:00:00

  • Liposomes in the treatment of infections.

    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

    authors: Bakker-Woudenberg IA,Storm G,Woodle MC

    更新日期:1994-01-01 00:00:00

  • α-Tocopherol as functional excipient for resveratrol and coenzyme Q10-loaded SNEDDS for improved bioavailability and prophylaxis of breast cancer.

    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

    authors: Jain S,Garg T,Kushwah V,Thanki K,Agrawal AK,Dora CP

    更新日期:2017-07-01 00:00:00

  • Tissue distribution and pulmonary targeting studies of cefpiramide sodium-loaded liposomes.

    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

    authors: Sun Q,Shi M,Shao W,Shi Y,Xi Y,Huang G

    更新日期:2011-01-01 00:00:00

  • Silica-deposited phospholipid nanotubules as a plausible drug targeting system.

    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

    authors: Kim I,Park YH,Rey DA,Batt CA

    更新日期:2008-11-01 00:00:00

  • SDS-coated atovaquone nanosuspensions show improved therapeutic efficacy against experimental acquired and reactivated toxoplasmosis by improving passage of gastrointestinal and blood-brain barriers.

    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

    authors: Shubar HM,Lachenmaier S,Heimesaat MM,Lohman U,Mauludin R,Mueller RH,Fitzner R,Borner K,Liesenfeld O

    更新日期:2011-02-01 00:00:00

  • Transfection of HEK cells via DNA-loaded PLGA and P(FASA) nanospheres.

    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

    authors: Sandor M,Mehta S,Harris J,Thanos C,Weston P,Marshall J,Mathiowitz E

    更新日期:2002-09-01 00:00:00

  • Oridonin-loaded poly(epsilon-caprolactone)-poly(ethylene oxide)-poly(epsilon-caprolactone) copolymer nanoparticles: preparation, characterization, and antitumor activity on mice with transplanted hepatoma.

    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

    authors: Feng N,Wu P,Li Q,Mei Y,Shi S,Yu J,Xu J,Liu Y,Wang Y

    更新日期:2008-07-01 00:00:00

  • Reduction of doxorubicin resistance in P-glycoprotein overexpressing cells by hybrid cell-penetrating and drug-binding peptide.

    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

    authors: Zheng Z,Aojula H,Clarke D

    更新日期:2010-07-01 00:00:00

  • Relationship between tumor cell load and sensitivity to the cytostatic effect of two novel platinum-bile acid complexes, Bamet-D3 and Bamet-UD2.

    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

    authors: Larena MG,Martinez-Diez MC,Macias RI,Dominguez MF,Serrano MA,Marin JJ

    更新日期:2002-08-01 00:00:00

  • Role of therapeutic agents on repolarisation of tumour-associated macrophage to halt lung cancer progression.

    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

    authors: Aldawsari HM,Gorain B,Alhakamy NA,Md S

    更新日期:2020-02-01 00:00:00

  • Prostate cancer cell-specific VEGF siRNA delivery system using cell targeting peptide conjugated polyplexes.

    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

    authors: Kim SH,Lee SH,Tian H,Chen X,Park TG

    更新日期:2009-05-01 00:00:00

  • Oral delivery of pathogens from the intestine to the nervous system.

    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

    authors: Baird AW,Campion DP,O'Brien L,Brayden DJ

    更新日期:2004-02-01 00:00:00

  • Gene delivery to brain cells with apoprotein E derived peptide conjugated to polylysine (apoEdp-PLL).

    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

    authors: Mousazadeh M,Palizban A,Salehi R,Salehi M

    更新日期:2007-04-01 00:00:00