Enhanced ganciclovir killing and bystander effect of human tumor cells transduced with a retroviral vector carrying a herpes simplex virus thymidine kinase gene mutant.

abstract：:Twenty-eight patients with advanced neovascular age-related macular degeneration (AMD) were given a single intravitreous injection of an E1-, partial E3-, E4-deleted adenoviral vector expressing human pigment epithelium- derived factor (AdPEDF.11). Doses ranging from 10(6) to 10(9.5) particle units (PU) were investiga...

journal_title：Human gene therapy

authors： Campochiaro PA,Nguyen QD,Shah SM,Klein ML,Holz E,Frank RN,Saperstein DA,Gupta A,Stout JT,Macko J,DiBartolomeo R,Wei LL

更新日期：2006-02-01 00:00:00

abstract：:We report on an antitumor treatment involving electrogene therapy (EGT), a newly developed in vivo gene transfer method using electroporation. We carried out in vivo EGT in a subcutaneous model of CT26 colon carcinoma cells, using plasmid DNAs encoding interleukin 12 (IL-12) subunits. For this purpose, we developed tw...

journal_title：Human gene therapy

authors： Tamura T,Nishi T,Goto T,Takeshima H,Dev SB,Ushio Y,Sakata T

更新日期：2001-07-01 00:00:00

abstract：:The addition of replication-defective recombinant adenovirus to plasmid transfection (termed here "adenofection") has been shown to increase plasmid transgene expression in limited studies. Similarly, the addition of cationic liposomes to adenovirus increases adenovirus-mediated gene transduction (termed here "lipoduc...

journal_title：Human gene therapy

authors： Dunphy EJ,Redman RA,Herweijer H,Cripe TP

更新日期：1999-09-20 00:00:00

abstract：:Liver ischemia-reperfusion (I/R) injury is a multifactorial process that affects graft function after liver transplantation. Inflammatory cytokines, such as tumor necrosis factor (TNF)-α, interleukin (IL)-6, IL-1β, and IL-18, have been shown to play key roles in the pathophysiology of liver I/R injury. Studies have in...

journal_title：Human gene therapy

authors： Zhu P,Duan L,Chen J,Xiong A,Xu Q,Zhang H,Zheng F,Tan Z,Gong F,Fang M

更新日期：2011-07-01 00:00:00

abstract：:Neurodegeneration in Parkinson's disease (PD) affects mainly dopaminergic neurons in the substantia nigra, where age-related, increasing percentages of cells lose detectable respiratory activity associated with depletion of intact mitochondrial DNA (mtDNA). Replenishment of mtDNA might improve neuronal bioenergetic fu...

journal_title：Human gene therapy

authors： Keeney PM,Quigley CK,Dunham LD,Papageorge CM,Iyer S,Thomas RR,Schwarz KM,Trimmer PA,Khan SM,Portell FR,Bergquist KE,Bennett JP Jr

更新日期：2009-08-01 00:00:00

abstract：:Gyrate atrophy is a progressive blindness associated with deficiency of ornithine aminotransferase (OAT). The strategy of using an autologous keratinocyte graft, modified to express high levels of OAT as an ornithine-catabolizing skin-based enzyme sink, is investigated. Two OAT-containing retroviral vectors were const...

journal_title：Human gene therapy

authors： Jensen TG,Sullivan DM,Morgan RA,Taichman LB,Nussenblatt RB,Blaese RM,Csaky KG

更新日期：1997-11-20 00:00:00

abstract：:Guidelines for testing gene therapy products for ecotropic replication-competent retrovirus (Eco-RCR) have not been delineated as they have for amphotropic viruses. To evaluate biologic assays that can detect these viruses, we compared an S(+)/L(-) assay and a marker rescue assay designed specifically for Eco-RCR dete...

journal_title：Human gene therapy

authors： Reeves L,Duffy L,Koop S,Fyffe J,Cornetta K

更新日期：2002-09-20 00:00:00

abstract：:Recombinant adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great potential for human gene therapy. One major challenge for translation of promising research to clinical development is the manufacture of sufficient quantities of AAV vectors that meet stringent standards for p...

journal_title：Human gene therapy

authors： Wright JF

更新日期：2009-07-01 00:00:00

abstract：:The enzyme hypoxanthine-guanine phosphoribosyltransferase (HGPRT) expressed by the parasite Trypanosoma brucei (Tb) can convert allopurinol, a purine analogue, to corresponding nucleotides with greater efficiency than its human homologue. We have developed a retroviral system that expresses the parasitic enzyme and te...

journal_title：Human gene therapy

authors： Trudeau C,Yuan S,Galipeau J,Benlimame N,Alaoui-Jamali MA,Batist G

更新日期：2001-09-01 00:00:00

abstract：:Isolated methylmalonic acidemia (MMA), a group of autosomal recessive inborn errors of metabolism, is most commonly caused by complete (mut(0)) or partial (mut(-)) deficiency of the enzyme methylmalonyl-CoA mutase (MUT). The severe metabolic instability and increased mortality experienced by many affected individuals,...

journal_title：Human gene therapy

authors： Harrington EA,Sloan JL,Manoli I,Chandler RJ,Schneider M,McGuire PJ,Calcedo R,Wilson JM,Venditti CP

更新日期：2016-05-01 00:00:00

abstract：:Gene therapy has evolved into a tempting strategy for the management of cancer and other life-threatening diseases. Various approaches employ retroviral vectors to deliver the therapeutic gene. The profound knowledge about retrovirus biology allows the generation of increasingly advanced vector systems as well as an a...

journal_title：Human gene therapy

authors： Brandtner EM,Kodajova P,Knapp E,Ertl R,Tabotta W,Salmons B,Günzburg WH,Hohenadl C

更新日期：2008-01-01 00:00:00

abstract：:The hepatocarcinoma-intestine-pancreas (HIP) gene, also called pancreatitis-associated protein-1 (PAP1) or Reg IIIalpha, is activated in most human hepatocellular carcinomas (HCCs) but not in normal liver, which suggests that HIP regulatory sequence could be used as efficient liver tumor-specific promoters to express ...

journal_title：Human gene therapy

authors： Hervé J,Cunha AS,Liu B,Valogne Y,Longuet M,Boisgard R,Brégerie O,Roux J,Guettier C,Calès P,Tavitian B,Samuel D,Clerc J,Bréchot C,Faivre J

更新日期：2008-09-01 00:00:00

abstract：:Gene therapy for Duchenne muscular dystrophy will likely require that the corrective dystrophin gene be delivered to a high fraction of muscle fibers in vivo. Because of the large size of the dystrophin cDNA, adenoviral (Ad) vectors have been developed for this application. However, Ad vectors transduce mature muscle ...

journal_title：Human gene therapy

authors： Menezes KM,Mok HS,Barry MA

更新日期：2006-03-01 00:00:00

abstract：:Williams-Beuren syndrome (WBS) and supravalvular aortic stenosis (SVAS) are genetic syndromes marked by the propensity to develop severe vascular stenoses. Vascular lesions in both syndromes are caused by haploinsufficiency of the elastin gene. We used these distinct genetic syndromes as models to evaluate the feasibi...

journal_title：Human gene therapy

authors： Zhang P,Huang A,Morales-Ruiz M,Starcher BC,Huang Y,Sessa WC,Niklason LE,Giordano FJ

更新日期：2012-11-01 00:00:00

abstract：:This study was designed to retrovirally transduce T cells by a protocol that would be simple, short, cost effective, applicable for clinical use, and efficient enough to avoid further selection of transduced T cells. Because retrovirally mediated infection is depending on the cell cycle, we first optimized the conditi...

journal_title：Human gene therapy

authors： Movassagh M,Boyer O,Burland MC,Leclercq V,Klatzmann D,Lemoine FM

更新日期：2000-05-20 00:00:00

abstract：:In previous studies we demonstrated that a modified human HSP70b promoter (HSE.70b) directs high levels of gene expression to tumor cells after mild hyperthermia treatment in the range of 41.5-44 degrees C. This transcriptional targeting system exhibits low basal activity at 37 degrees C, is highly induced (950-fold) ...

journal_title：Human gene therapy

authors： Brade AM,Szmitko P,Ngo D,Liu FF,Klamut HJ

更新日期：2003-03-20 00:00:00

abstract：:Stem cell mobilization to injured tissue contributes to neovascularization, resulting in regeneration after myocardial infarction (MI). We previously showed that direct cardiac injection of a recombinant lentivirus (LV) that engineers expression of membrane-bound stem cell factor (mSCF) improves outcomes immediately a...

journal_title：Human gene therapy

authors： Sun Z,Lee CJ,Mejia-Guerrero S,Zhang Y,Higuchi K,Li RK,Medin JA

更新日期：2012-12-01 00:00:00

abstract：:To achieve effective gene therapy, it is necessary to selectively and efficiently transfect therapeutic gene into targeted cells. In this study, we developed a combination method using mannosylated lipoplexes, which show selectivity to antigen-presenting cells such as macrophages and dendritic cells, and bubble liposo...

journal_title：Human gene therapy

authors： Un K,Kawakami S,Suzuki R,Maruyama K,Yamashita F,Hashida M

更新日期：2010-01-01 00:00:00

abstract：:Recombinant adeno-associated virus serotype 2 (rAAV2)-based human gene therapy for cystic fibrosis has progressed through a series of preclinical studies and phase I and II clinical trials. This agent has shown an encouraging safety profile, consistent levels of DNA transfer, and positive evidence of short-term clinic...

journal_title：Human gene therapy

authors： Flotte TR,Schwiebert EM,Zeitlin PL,Carter BJ,Guggino WB

更新日期：2005-08-01 00:00:00

abstract：:Replication-deficient adenoviruses are known to induce acute injury and inflammation of infected tissues, thus limiting their use for human gene therapy. However, molecular mechanisms triggering this response have not been fully defined. To characterize this response, chemokine expression was evaluated in DBA/2 mice f...

journal_title：Human gene therapy

authors： Muruve DA,Barnes MJ,Stillman IE,Libermann TA

更新日期：1999-04-10 00:00:00

abstract：:Despite improvements in drug and device therapy for heart failure, hospitalization rates and mortality have changed little in the past decade. Randomized clinical trials using gene transfer to improve function of the failing heart are the focus of this review. Four randomized clinical trials of gene transfer in heart ...

journal_title：Human gene therapy

authors： Penny WF,Hammond HK

更新日期：2017-05-01 00:00:00

abstract：:Baculovirus vectors recently have been shown to be capable of efficient transduction of human hepatoma cells and primary hepatocytes in culture. This paper describes the generation of a novel recombinant baculovirus (VGZ3) in which the vesicular stomatitis virus glycoprotein G (VSV G) is present in the viral envelope....

journal_title：Human gene therapy

authors： Barsoum J,Brown R,McKee M,Boyce FM

更新日期：1997-11-20 00:00:00

abstract：:One of the major obstacles to pulmonary-directed gene therapy using adenoviral vectors is the induction of inflammation. We investigated whether the adenoviral particles that constitute the initial inoculum can serve as an inflammatory stimulus, independent of their ability to express genes that they contain. Viral pa...

journal_title：Human gene therapy

authors： McCoy RD,Davidson BL,Roessler BJ,Huffnagle GB,Janich SL,Laing TJ,Simon RH

更新日期：1995-12-01 00:00:00

abstract：:Adenovirus-polylysine-DNA complexes were evaluated for their capacity to accomplish direct in vivo gene transfer to airway epithelium employing a rodent model. Binary complexes containing transferrin or adenovirus, or combination complexes containing both transferrin and adenovirus, were evaluated. The highest in vitr...

journal_title：Human gene therapy

authors： Gao L,Wagner E,Cotten M,Agarwal S,Harris C,Rømer M,Miller L,Hu PC,Curiel D

更新日期：1993-02-01 00:00:00

abstract：:Umbilical cord blood (CB) from the early gestational human fetus is recognized as a rich source of hematopoietic stem cells. To examine the value of fetal CB for gene therapy of inborn immunohematopoietic disorders, we tested the feasibility of genetic modification of CD34(+) cells from CB at weeks 24 to 34 of pregnan...

journal_title：Human gene therapy

authors： Luther-Wyrsch A,Costello E,Thali M,Buetti E,Nissen C,Surbek D,Holzgreve W,Gratwohl A,Tichelli A,Wodnar-Filipowicz A

更新日期：2001-03-01 00:00:00

abstract：:The rapid inactivation of murine-derived retroviral vectors in human or nonhuman primate sera is largely attributed to the activity of complement mediated through the classical pathway. In this study, we have further investigated the relationship between the human complement cascade and retrovirus inactivation. Preinc...

journal_title：Human gene therapy

authors： Rother RP,Squinto SP,Mason JM,Rollins SA

更新日期：1995-04-01 00:00:00

abstract：:Here we show potent inhibition of HIV-1 replication in a human T cell line and primary human CD4(+) cells by expressing a single antiviral protein. Nullbasic is a mutant form of the HIV-1 Tat protein that was previously shown to strongly inhibit HIV-1 replication in nonhematopoietic cell lines by targeting three steps...

journal_title：Human gene therapy

authors： Apolloni A,Lin MH,Sivakumaran H,Li D,Kershaw MH,Harrich D

更新日期：2013-03-01 00:00:00

abstract：:Heme oxygenase 1 (HO-1) is an inducible enzyme that catalyzes heme to generate bilirubin, ferritin, and carbon monoxide. Because enhanced expression of HO-1 confers protection against many types of cell and tissue damage by modulating apoptotic cell death or cytokine expression profiles, we hypothesized that adenoviru...

journal_title：Human gene therapy

authors： Tsuburai T,Suzuki M,Nagashima Y,Suzuki S,Inoue S,Hasiba T,Ueda A,Ikehara K,Matsuse T,Ishigatsubo Y

更新日期：2002-11-01 00:00:00

abstract：:We report a novel method for targeting adenovirus-mediated gene delivery. By irradiating mammalian cells prior to adenoviral transduction, adenoviral gene transfer is greatly improved and the adenoviral genome integrates into cellular DNA. In this work, human and rodent cell lines were irradiated and subsequently tran...

journal_title：Human gene therapy

authors： Zeng M,Cerniglia GJ,Eck SL,Stevens CW

更新日期：1997-06-10 00:00:00

abstract：:Recombinant poxviruses expressing immunomodulatory molecules together with specific antigens represent powerful vaccines for cancer immunotherapy. Recently, we and others have demonstrated, in vitro and in vivo, that coexpression of CD80 and CD86 costimulatory molecules enhances the immunogenic capacity of a recombina...

journal_title：Human gene therapy

authors： Feder-Mengus C,Schultz-Thater E,Oertli D,Marti WR,Heberer M,Spagnoli GC,Zajac P

更新日期：2005-03-01 00:00:00