Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer.

abstract：:We have developed a retroviral vector coexpressing the multidrug-resistance 1 (MDR1) cDNA for inducing cancer drug resistance and the truncated version of the low-affinity nerve growth factor receptor (DeltaLNGFR) for cell-surface marking of transduced cells. The vector is based on the FMEV backbone which mediates hig...

journal_title：Gene therapy

authors： Hildinger M,Schilz A,Eckert HG,Bohn W,Fehse B,Zander A,Ostertag W,Baum C

更新日期：1999-07-01 00:00:00

abstract：:Short hairpin RNA (shRNA)-mediated gene regulation is a commonly used technique for gene manipulation. An efficient and safe delivery system is indispensable when shRNA is delivered into living organisms for gene therapy. Previous studies have proved that DNA and protein can be delivered into dendritic cells (DCs) by ...

journal_title：Gene therapy

authors： Zhang L,Zhang T,Wang L,Shao S,Chen Z,Zhang Z

更新日期：2014-07-01 00:00:00

abstract：:Virotherapy is currently being developed for many different types of viruses including replication-competent murine leukaemia virus (MLV) as a novel tool in cancer therapy. However, there is the risk of insertional mutagenesis associated with this virus, making careful preclinical studies necessary before its first ap...

journal_title：Gene therapy

authors： Duerner LJ,Schwantes A,Schneider IC,Cichutek K,Buchholz CJ

更新日期：2008-11-01 00:00:00

abstract：:The pressing challenge for contemporary gene therapy is to deliver enough therapeutic genes to enough cancer cells in vivo. With the aim of improving viral distribution and tumor penetration, we explored the use of decorin to enhance viral spreading and tumor tissue penetration. We generated decorin-expressing replica...

journal_title：Gene therapy

authors： Choi IK,Lee YS,Yoo JY,Yoon AR,Kim H,Kim DS,Seidler DG,Kim JH,Yun CO

更新日期：2010-02-01 00:00:00

abstract：:To facilitate the understanding of the complex process of target gene expression and its control, we report a modified inducible system for activation or repression of target gene expression in response to an exogenously administered compound. The main component of this inducible system is a chimeric transcriptional a...

journal_title：Gene therapy

authors： Wang Y,Xu J,Pierson T,O'Malley BW,Tsai SY

更新日期：1997-05-01 00:00:00

abstract：:Mutations in GJB2, which codes for the gap junction (GJ) protein connexin26 (Cx26), are the most common causes of human nonsyndromic hereditary deafness. We inoculated modified adeno-associated viral (AAV) vectors into the scala media of early postnatal conditional Gjb2 knockout mice to drive exogenous Cx26 expression...

journal_title：Gene therapy

authors： Yu Q,Wang Y,Chang Q,Wang J,Gong S,Li H,Lin X

更新日期：2014-01-01 00:00:00

abstract：:Oligonucleotides (ODNs) show great promise in their ability to specifically inhibit single gene expression but must cross the cell membrane, escape the endosomal vesicle, and possibly traverse the nuclear membrane to arrive at their intracellular target molecules. In an attempt to improve the delivery of phosphodieste...

journal_title：Gene therapy

authors： Ebbinghaus SW,Vigneswaran N,Miller CR,Chee-Awai RA,Mayfield CA,Curiel DT,Miller DM

更新日期：1996-04-01 00:00:00

abstract：:The canine is the most important large animal model for testing novel hemophilia A (HA) treatment. It is often necessary to use canine factor VIII (cFIII) gene or protein for the evaluation of HA treatment in the canine model. However, different biological properties between cFVIII and human FVIII (hFVIII) indicated t...

journal_title：Gene therapy

authors： Wang Q,Dong B,Firrman J,Wu W,Roberts S,Moore AR,Liu LS,Chin MP,Diao Y,Kost J,Xiao W

更新日期：2016-07-01 00:00:00

abstract：:An amendment to this paper has been published and can be accessed via a link at the top of the paper. ...

journal_title：Gene therapy

authors： Lan Y,Tao Y,Wang Y,Ke J,Yang Q,Liu X,Su B,Wu Y,Lin CP,Zhong G

更新日期：2020-08-01 00:00:00

abstract：:Exogenous osteoprotegerin (OPG) gene modification appears a therapeutic strategy for osteolytic aseptic loosening. The feasibility and efficacy of a cell-based OPG gene delivery approach were investigated using a murine model of knee prosthesis failure. A titanium pin was implanted into mouse proximal tibia to mimic a...

journal_title：Gene therapy

authors： Zhang L,Jia TH,Chong AC,Bai L,Yu H,Gong W,Wooley PH,Yang SY

更新日期：2010-10-01 00:00:00

abstract：:Cationic lipid-based delivery systems such as lipoplexes or stabilized plasmid-lipid particles (SPLP) represent a safer alternative to viral systems for gene therapy applications. We studied the impact of cell cycle status on the efficiency of transfection of human ovarian carcinoma tumor cells using two cationic-lipi...

journal_title：Gene therapy

authors： Mortimer I,Tam P,MacLachlan I,Graham RW,Saravolac EG,Joshi PB

更新日期：1999-03-01 00:00:00

abstract：:The E1 deleted adenoviral vectors are efficient at gene transfer to cells in culture or in animals. However, their use is limited because of an immune-mediated loss of transduced cells. This immune response is believed to result from low-level production of viral antigens from these vectors after gene transfer. The ea...

journal_title：Gene therapy

authors： Scaria A,Curiel DT,Kay MA

更新日期：1995-06-01 00:00:00

abstract：:To test whether fast-acting, self-complimentary (sc), adeno-associated virus-mediated RPE65 expression prevents cone degeneration and/or restores cone function, we studied two mouse lines: the Rpe65-deficient rd12 mouse and the Rpe65-deficient, rhodopsin null ('that is, cone function-only') Rpe65(-/-)::Rho(-/-) mouse....

journal_title：Gene therapy

authors： Pang J,Boye SE,Lei B,Boye SL,Everhart D,Ryals R,Umino Y,Rohrer B,Alexander J,Li J,Dai X,Li Q,Chang B,Barlow R,Hauswirth WW

更新日期：2010-07-01 00:00:00

abstract：:We explored the possibility of using a genetic approach to inhibit integrin-mediated endothelial cell adhesion and survival. We constructed recombinant adenoviruses (Ads) expressing chimeric proteins consisting of the cytoplasmic and transmembrane domains of integrin beta1 (CH1), beta3 (CH3) or the beta1 transmembrane...

journal_title：Gene therapy

authors： Oguey D,George PW,Rüegg C

更新日期：2000-08-01 00:00:00

abstract：:The GM2-gangliosidoses are neurological diseases causing premature death, thus developing effective treatment protocols is urgent. GM2-gangliosidoses result from deficiency of a lysosomal enzyme β-hexosaminidase (Hex) and subsequent accumulation of GM2 gangliosides. Genetic changes in HEXA, encoding the Hex α subunit,...

journal_title：Gene therapy

authors： Ou L,Przybilla MJ,Tăbăran AF,Overn P,O'Sullivan MG,Jiang X,Sidhu R,Kell PJ,Ory DS,Whitley CB

更新日期：2020-05-01 00:00:00

abstract：:Human adenovirus (Ad) vectors are extensively used as gene transfer vehicles. However, a serious obstacle for the use of these vectors in clinical applications is due to pre-existing immunity to human Ads affecting the efficacy of gene transfer. One of the approaches to circumvent host immune response could be the dev...

journal_title：Gene therapy

authors： Logunov DY,Ilyinskaya GV,Cherenova LV,Verhovskaya LV,Shmarov MM,Chumakov PM,Kopnin BP,Naroditsky BS

更新日期：2004-01-01 00:00:00

abstract：:We have comparatively evaluated the efficiency of a series of retroviral vectors transducing the gp91-phox gene, whose defects are responsible for impaired production of superoxide anion (O2-) by phagocytic cells and lead to the X-linked form of chronic granulomatous disease (X-CGD). These vectors included four constr...

journal_title：Gene therapy

authors： Zentilin L,Qin G,Tafuro S,Dinauer MC,Baum C,Giacca M

更新日期：2000-01-01 00:00:00

abstract：:Inadequate antiprotease activity in the lungs due to alpha-1-antitrypsin (A1AT) deficiency is a factor of early-onset emphysema. We propose a new approach to gene therapy that involves the intratracheal delivery of macrophages expressing human A1AT (hA1AT). Recombinant adeno-associated virus (rAAV) plasmids encoding t...

journal_title：Gene therapy

authors： Zhang D,Wu M,Nelson DE,Pasula R,Martin WJ 2nd

更新日期：2003-12-01 00:00:00

abstract：:RNA interference (RNAi) is an evolutionarily conserved process by which plants and animals protect their genomes utilizing small, double-stranded RNAs to degrade target RNAs in a sequence-specific manner. Post-transcriptional gene silencing by these moieties can lead to degradation of both cellular and viral RNAs. It ...

journal_title：Gene therapy

authors： Zhou N,Fang J,Mukhtar M,Acheampong E,Pomerantz RJ

更新日期：2004-12-01 00:00:00

abstract：:The efficiency of plasmid gene transfer to skeletal muscle can be significantly improved by the application of an electrical field to the muscle following injection of plasmid DNA. However, this electrotransfer is associated with significant muscle damage which may result in substantial loss of transfected muscle fibr...

journal_title：Gene therapy

authors： McMahon JM,Signori E,Wells KE,Fazio VM,Wells DJ

更新日期：2001-08-01 00:00:00

abstract：:Polyethylenimine (PEI) has been studied as an efficient nonviral gene transfer vector. Here, we report the biodistribution fates and safety of plasmid DNA intravenously administered in PEI complexes. Using pCMVbeta as a model gene, the biodistribution of plasmid DNA was measured by quantitative polymerase chain reacti...

journal_title：Gene therapy

authors： Oh YK,Kim JP,Yoon H,Kim JM,Yang JS,Kim CK

更新日期：2001-10-01 00:00:00

abstract：:The majority of immunotherapy-based gene therapy protocols consist of ex vivo gene transfer in tumor cells. To prevent further in vivo growth, modified cells must be irradiated before reinjection into patients. The present study examines the effects of gamma-irradiation on transgene expression in transduced leukemic c...

journal_title：Gene therapy

authors： Vereecque R,Saudemont A,Wickham TJ,Gonzalez R,Hetuin D,Fenaux P,Quesnel B

更新日期：2003-02-01 00:00:00

abstract：:RNAi represents a powerful technology to specifically downregulate the expression of target genes. For cancer research and therapy, an efficient in vivo delivery system is supposed to distribute RNAi to all tumour cells upon systemic administration. We present replication-competent murine leukaemia virus (MLV) vectors...

journal_title：Gene therapy

authors： Schaser T,Wrede C,Duerner L,Sliva K,Cichutek K,Schnierle B,Buchholz CJ

更新日期：2011-10-01 00:00:00

abstract：:Glucagon-like peptide 1 (GLP-1) and its analogue exendin-4 (Ex4) have displayed potent glucose homeostasis-modulating characteristics in type 2 diabetes (T2D). However, there are few reports of effectiveness in type 1 diabetes (T1D) therapy, where there is massive loss of beta cells. We previously described a novel GL...

journal_title：Gene therapy

authors： Soltani N,Kumar M,Glinka Y,Prud'homme GJ,Wang Q

更新日期：2007-06-01 00:00:00

abstract：:Transplantation of allogeneic cells as well as of genetically corrected autologous cells are potent approaches to restore cellular functions in patients suffering from genetic diseases. The recipient's immune responses against non-self-antigens may compromise the survival of the grafted cells. Recipients of the graft ...

journal_title：Gene therapy

authors： Bots STF,Hoeben RC

更新日期：2017-07-01 00:00:00

abstract：:We have used a particular folate receptor, which is overexpressed in tumor cells, for targeted DNA delivery into these cell types. This folate receptor internalizes folate through caveolae by a process named potocytosis, which is distinct from endocytosis, through clathrin-coated pits. When folate conjugated to poly-L...

journal_title：Gene therapy

authors： Gottschalk S,Cristiano RJ,Smith LC,Woo SL

更新日期：1994-05-01 00:00:00

abstract：:Inhibition of virus replication by means of RNA interference has been reported for several important human pathogens, including human immunodeficiency virus type 1 (HIV-1). RNA interference against these pathogens has been accomplished by introduction of virus-specific synthetic small interfering RNAs (siRNAs) or DNA ...

journal_title：Gene therapy

authors： Konstantinova P,de Vries W,Haasnoot J,ter Brake O,de Haan P,Berkhout B

更新日期：2006-10-01 00:00:00

abstract：:This study was designed to evaluate the utility of positron emission tomography (PET) to quantify the magnitude and spatial distribution of transgene expression after different methods of adenoviral vector delivery (with surfactant- and saline-based vehicles) within rat lungs. In all, 17 animals (eight in the surfacta...

journal_title：Gene therapy

authors： Richard JC,Factor P,Welch LC,Schuster DP

更新日期：2003-12-01 00:00:00

abstract：:With the long-term goal of developing a gene-based treatment for osteoarthritis (OA), we performed studies to evaluate the equine joint as a model for adeno-associated virus (AAV)-mediated gene transfer to large, weight-bearing human joints. A self-complementary AAV2 vector containing the coding regions for human inte...

journal_title：Gene therapy

authors： Watson RS,Broome TA,Levings PP,Rice BL,Kay JD,Smith AD,Gouze E,Gouze JN,Dacanay EA,Hauswirth WW,Nickerson DM,Dark MJ,Colahan PT,Ghivizzani SC

更新日期：2013-06-01 00:00:00

abstract：:Hepatic sequestration of systemically administered adenoviral vectors reduces the number of viral particles available for delivery to other tissues. The biological basis of this phenomenon was investigated using a new in vivo technique which permitted imaging in real time. Recombinant adenovirus serotype 5 knob (Ad5K)...

journal_title：Gene therapy

authors： Zinn KR,Douglas JT,Smyth CA,Liu HG,Wu Q,Krasnykh VN,Mountz JD,Curiel DT,Mountz JM

更新日期：1998-06-01 00:00:00