Characterization of plasmid DNA transfer into mouse skeletal muscle: evaluation of uptake mechanism, expression and secretion of gene products into blood.

abstract：:Insulin gene therapy in clinical medicine is currently hampered by the inability to regulate insulin secretion in a physiological manner, the inefficiency with which the gene is delivered, and the short duration of gene expression. To address these issues, we injected the liver of streptozotocin-induced diabetic rats ...

journal_title：Gene therapy

authors： Kim YD,Park KG,Morishita R,Kaneda Y,Kim SY,Song DK,Kim HS,Nam CW,Lee HC,Lee KU,Park JY,Kim BW,Kim JG,Lee IK

更新日期：2006-02-01 00:00:00

abstract：:Genetic engineering of T lymphocytes for adoptive clinical immunotherapy calls for efficient gene transduction methods. Therefore, a transient retroviral gene transduction system 'STITCH' was developed comprising pSTITCH retroviral vector encoding the transgene, plasmids encoding Moloney murine leukemia virus gag/pol ...

journal_title：Gene therapy

authors： Weijtens ME,Willemsen RA,Hart EH,Bolhuis RL

更新日期：1998-09-01 00:00:00

abstract：:Hydrodynamic gene delivery is an attractive option for non-viral liver gene therapy, but requires evaluation of efficacy, safety and clinically applicable techniques in large animal models. We have evaluated retrograde delivery of DNA to the whole liver via the isolated segment of inferior vena cava (IVC) draining the...

journal_title：Gene therapy

authors： Fabre JW,Grehan A,Whitehorne M,Sawyer GJ,Dong X,Salehi S,Eckley L,Zhang X,Seddon M,Shah AM,Davenport M,Rela M

更新日期：2008-03-01 00:00:00

abstract：:Abnormal excitation-contraction coupling is a key pathophysiologic component of heart failure (HF), and at a molecular level reduced expression of the sarcoplasmic reticulum (SR) Ca(2+) ATPase (SERCA2a) is a major contributor. Previous studies in small animals have suggested that restoration of SERCA function is benef...

journal_title：Gene therapy

authors： Byrne MJ,Power JM,Preovolos A,Mariani JA,Hajjar RJ,Kaye DM

更新日期：2008-12-01 00:00:00

abstract：:Morphine appears to be the most active metabolite of heroin; therefore, the effects of morphine are important in understanding the ramifications of heroin abuse. Opioid physical dependence (withdrawal response) may have very long-lasting effects on the motivation for reward, including the incubation of cue-induced dru...

journal_title：Gene therapy

authors： Iida T,Yi H,Liu S,Ikegami D,Zheng W,Liu Q,Takahashi K,Kashiwagi Y,Goins WF,Glorioso JC,Hao S

更新日期：2017-05-01 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：:Acute lung injury (ALI) and acute respiratory distress syndrome (ARDS) are associated with diverse disorders and characterized by disruption of the alveolar-capillary barrier, leakage of edema fluid into the lung, and substantial inflammation leading to acute respiratory failure. Gene therapy is a potentially powerful...

journal_title：Gene therapy

authors： Lin X,Barravecchia M,Kothari P,Young JL,Dean DA

更新日期：2016-06-01 00:00:00

abstract：:Foamy virus (FV) vectors are promising for hematopoietic stem cell (HSC) gene therapy but preclinical data on the clonal composition of FV vector-transduced human repopulating cells is needed. Human CD34(+) human cord blood cells were transduced with an FV vector encoding a methylguanine methyltransferase (MGMT)P140K ...

journal_title：Gene therapy

authors： Olszko ME,Adair JE,Linde I,Rae DT,Trobridge P,Hocum JD,Rawlings DJ,Kiem HP,Trobridge GD

更新日期：2015-07-01 00:00:00

abstract：:Vessel wall inflammation and matrix destruction are critical to abdominal aortic aneurysm (AAA) formation and rupture. We have previously shown that urokinase plasminogen activator (uPA) is highly expressed in experimental AAA and is essential for AAA formation and expansion. In this study, we examined the effects of ...

journal_title：Gene therapy

authors： Qian HS,Gu JM,Liu P,Kauser K,Halks-Miller M,Vergona R,Sullivan ME,Dole WP,Deng GG

更新日期：2008-02-01 00:00:00

abstract：:Apo(a) is a very atherogenic plasma protein without apparent function, which is highly expressed in humans. The variation in plasma Lp(a) concentration among individuals is considerable. Approximately 10-15% of the white population exhibit plasma Lp(a) concentrations above the atherogenic cut-off value of approximatel...

journal_title：Gene therapy

authors： Frank S,Gauster M,Strauss J,Hrzenjak A,Kostner GM

更新日期：2001-03-01 00:00:00

abstract：:The failure of pharmacological approaches to cure infection with the human immunodeficiency virus (HIV) has renewed the interest in gene-based therapies. Among the various strategies that are currently explored, the blockade of HIV entry into susceptible T cells and macrophages promises to be the most powerful interve...

journal_title：Gene therapy

authors： Schambach A,Schiedlmeier B,Kühlcke K,Verstegen M,Margison GP,Li Z,Kamino K,Bohne J,Alexandrov A,Hermann FG,von Laer D,Baum C

更新日期：2006-07-01 00:00:00

abstract：:We have compared the efficacy of daily injection of recombinant leptin protein (rh-leptin) with adenovirus-mediated delivery of the murine or human leptin gene (Ad-leptin) for treatment of obesity in the obese (ob/ob) mouse model. We demonstrate an improved correction profile for obesity and associated surrogate marke...

journal_title：Gene therapy

authors： Morsy MA,Gu MC,Zhao JZ,Holder DJ,Rogers IT,Pouch WJ,Motzel SL,Klein HJ,Gupta SK,Liang X,Tota MR,Rosenblum CI,Caskey CT

更新日期：1998-01-01 00:00:00

abstract：:For many gene therapy applications the effective titre of retroviral vectors is a limiting factor both in vitro and in vivo. Purification and concentration of retrovirus from packaging cell supernatant can overcome this problem. To this end we have investigated a novel procedure which involves complexing retrovirus to...

journal_title：Gene therapy

authors： Darling D,Hughes C,Galea-Lauri J,Gäken J,Trayner ID,Kuiper M,Farzaneh F

更新日期：2000-06-01 00:00:00

abstract：:A retroviral vector constructed from the murine leukemia virus (MLV) can only express transgenes in cells undergoing mitosis, indicating its suitability as a delivery vehicle for cancer gene therapy. However, the transduction efficiency (TE) of retroviruses embedding endogenous envelope proteins in human cancer cells ...

journal_title：Gene therapy

authors： Lee H,Song JJ,Kim E,Yun CO,Choi J,Lee B,Kim J,Chang JW,Kim JH

更新日期：2001-02-01 00:00:00

abstract：:HaCaT cells, a spontaneously immortalised, nontumorigenic keratinocyte line, were used as a more amenable model than primary keratinocytes for ex vivo-mediated gene transfer. These cells were transduced with retroviral vectors containing the factor IX cDNA under the control of a cytomegaloviral (CMV) promoter/enhancer...

journal_title：Gene therapy

authors： Page SM,Brownlee GG

更新日期：1998-03-01 00:00:00

abstract：:Although particle-mediated gene transfer using gene gun technology has been applied for gene transfer into epidermis, applications of this technology to visceral tissues have not been well investigated. Although all helium gas-driven gene gun instruments have used macrocarriers to discharge DNA-coated microprojectiles...

journal_title：Gene therapy

authors： Kuriyama S,Mitoro A,Tsujinoue H,Nakatani T,Yoshiji H,Tsujimoto T,Yamazaki M,Fukui H

更新日期：2000-07-01 00:00:00

abstract：:Adenovirus-mediated gene transfer of interferon gamma (AdIFN) elicits rejection of intracerebral Lewis lung carcinoma. In this system, gene transfer into brain parenchymal cells is both necessary and sufficient to generate the antitumor response. Despite persistent parenchymal inflammation and demyelination, wild-type...

journal_title：Gene therapy

authors： Fathallah-Shaykh HM,Kafrouni AI,Zhao LJ,Diaz-Arrastia R,Garcia JA,Frawley WH,Forman J

更新日期：2000-12-01 00:00:00

abstract：:Several groups are assessing the use of cationic lipids for respiratory gene therapy. To date no human data are available regarding the safety of intra-pulmonary cationic lipid delivery. In preparation for a trial of pulmonary delivery of the CFTR gene, we have assessed the safety of nebulised lipid GL-67/DOPE/DMPE-PE...

journal_title：Gene therapy

authors： Chadwick SL,Kingston HD,Stern M,Cook RM,O'Connor BJ,Lukasson M,Balfour RP,Rosenberg M,Cheng SH,Smith AE,Meeker DP,Geddes DM,Alton EW

更新日期：1997-09-01 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：: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：:Advances in genetic analysis and a greater understanding of human immunodeficiency virus (HIV) molecular pathogenesis have identified critical viral targets for gene interference strategies. RNase P molecules have been proposed as a novel approach for gene targeting based upon their potent catalytic activity, as well ...

journal_title：Gene therapy

authors： Hnatyszyn H,Spruill G,Young A,Seivright R,Kraus G

更新日期：2001-12-01 00:00:00

abstract：:Defensins are small cationic proteins that harbor broad-spectrum microbicidal activity against bacteria, fungi and viruses. This study examines the effects on pathogens of the epidermis engineered to express human beta-defensin 3 (HBD3) to combat bacterial infections. First, we examined the localization of HBD3 in the...

journal_title：Gene therapy

authors： Sawamura D,Goto M,Shibaki A,Akiyama M,McMillan JR,Abiko Y,Shimizu H

更新日期：2005-05-01 00:00:00

abstract：:Lentiviral vectors pseudotyped with the baculovirus envelope protein GP64 transduce primary cultures of human airway epithelia (HAE) at their apical surface. Our goal in this study was to harness a directed evolution approach to develop a novel envelope glycoprotein with increased transduction properties for HAE. Usin...

journal_title：Gene therapy

authors： Sinn PL,Hwang BY,Li N,Ortiz JLS,Shirazi E,Parekh KR,Cooney AL,Schaffer DV,McCray PB Jr

更新日期：2017-10-01 00:00:00

abstract：:The progression of chronic renal diseases is considered as an irreversible process that eventually leads to end-stage renal failure characterized by extensive tissue fibrosis. At present, chronic renal fibrosis is incurable and the incidence of affected patients is on the rise worldwide. In this study, we demonstrate ...

journal_title：Gene therapy

authors： Yang J,Dai C,Liu Y

更新日期：2001-10-01 00:00:00

abstract：:Sustainable correction of severe human genetic disorders of self-renewing tissues, such as the blistering skin disease junctional epidermolysis bullosa (JEB), is facilitated by stable genomic integration of therapeutic genes into somatic tissue stem cells. While integrating viral vectors can achieve this, they suffer ...

journal_title：Gene therapy

authors： Ortiz-Urda S,Lin Q,Yant SR,Keene D,Kay MA,Khavari PA

更新日期：2003-07-01 00:00:00

abstract：:Signal transducer and activator of transcription 3 (STAT3) is constitutively activated in diverse cancers, which contributes to the proliferation and survival of cancer cells by upregulating apoptosis inhibitors and cell cycle regulators. Suppressor of cytokine signaling 1 (SOCS1) is an important negative regulator of...

journal_title：Gene therapy

authors： Liu L,Li W,Wei X,Cui Q,Lou W,Wang G,Hu X,Qian C

更新日期：2013-01-01 00:00:00

abstract：:There is growing interest in gene delivery to the eye in order to develop gene therapy for the many ocular disorders which may be amenable to this approach. To date, recombinant adenoviruses (AV) have been the main vector used for gene delivery to anterior and posterior segments in animal models. As with delivery to o...

journal_title：Gene therapy

authors： Ali RR,Reichel MB,Byrnes AP,Stephens CJ,Thrasher AJ,Baker D,Hunt DM,Bhattacharya SS

更新日期：1998-11-01 00:00:00

abstract：:A highly desirable feature for an human immunodeficiency virus type 1 (HIV-1) vaccine is the ability to induce broadly reactive anti-envelope antibodies that can neutralize primary HIV-1 isolates. Two immunizations with an HIV-1 envelope-encoding plasmid together with recombinant granulocyte-macrophage colony-stimulat...

journal_title：Gene therapy

authors： Rollman E,Hinkula J,Arteaga J,Zuber B,Kjerrström A,Liu M,Wahren B,Ljungberg K

更新日期：2004-07-01 00:00:00

abstract：:Earlier this laboratory constructed a herpes simplex virus 1 recombinant (R5111) that carries a IL13 ligand inserted into glycoprotein D and can enter cells via the IL13Ralpha2 receptor commonly expressed on the surface of malignant glioma cells. In this report, we describe the properties of two recombinant viruses ca...

journal_title：Gene therapy

authors： Kamiyama H,Zhou G,Roizman B

更新日期：2006-04-01 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