An adenoviral vector-based mucosal vaccine is effective in protection against botulism.

abstract：:Angiogenesis plays a pivotal role in tumor growth, tissue invasion and metastasis. Endostatin is an angiogenesis inhibitor and has been shown to reduce tumor growth in animal models. However, therapy with recombinant endostatin protein was hampered by its short half-life and very-low yield of bioactive protein. We per...

journal_title：Gene therapy

authors： Li HL,Li S,Shao JY,Lin XB,Cao Y,Jiang WQ,Liu RY,Zhao P,Zhu XF,Zeng MS,Guan ZZ,Huang W

更新日期：2008-02-01 00:00:00

abstract：:Catheter-based percutaneous transluminal gene delivery (PTGD) into the coronary artery still falls behind the expectations of an efficient myocardial gene delivery system. In this study gene delivery was applied by selective pressure-regulated retroinfusion through the coronary veins to prolong adhesion of replication...

journal_title：Gene therapy

authors： Boekstegers P,von Degenfeld G,Giehrl W,Heinrich D,Hullin R,Kupatt C,Steinbeck G,Baretton G,Middeler G,Katus H,Franz WM

更新日期：2000-02-01 00:00:00

abstract：:Isolated limb perfusion (ILP) is a limb salvage surgical modality used to deliver chemotherapy and biologic agents to locally advanced and recurrent extremity soft tissue sarcoma (STS), and may be readily tailored for delivery of gene therapy. We set out to test the feasibility of delivering AdFLAGp53 (replication inc...

journal_title：Gene therapy

authors： Hannay J,Davis JJ,Yu D,Liu J,Fang B,Pollock RE,Lev D

更新日期：2007-04-01 00:00:00

abstract：:We report on systemic delivery and long-term biological effects of apolipoprotein E (apoE) obtained by intramuscular (i.m.) plasmid DNA injection. ApoE plays an important role in lipoprotein catabolism and apoE knock-out mice develop severe hypercholesterolemia and diffuse atherosclerosis. We have injected apoE-defici...

journal_title：Gene therapy

authors： Rinaldi M,Catapano AL,Parrella P,Ciafrè SA,Signori E,Seripa D,Uboldi P,Antonini R,Ricci G,Farace MG,Fazio VM

更新日期：2000-11-01 00:00:00

abstract：:Self-amplifying RNA (saRNA) is a promising biotherapeutic tool that has been used as a vaccine against both infectious diseases and cancer. saRNA has been shown to induce protein expression for up to 60 days and elicit immune responses with lower dosing than messenger RNA (mRNA). Because saRNA is a large (~9500 nt), n...

journal_title：Gene therapy

authors： Blakney AK,McKay PF,Yus BI,Aldon Y,Shattock RJ

更新日期：2019-09-01 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：:Cellular immunity against cancer can be achieved with viral vector- and DNA-based immunizations. In preclinical studies, cancer vaccines are very potent, but in clinical trials these potencies are not achieved yet. Thus, a rational approach to improve cancer vaccines is warranted. We previously demonstrated that the r...

journal_title：Gene therapy

authors： Ip PP,Boerma A,Walczak M,Oosterhuis K,Haanen JB,Schumacher TN,Nijman HW,Daemen T

更新日期：2015-07-01 00:00:00

abstract：:Evidence has documented the tumor-promoting properties of long non-coding RNA (lncRNA) FOXD2 adjacent opposite strand RNA 1 (FOXD2-AS1) in many cancers. However, little is known about its role in gallbladder cancer. Here, we aimed to characterize the functional relevance of lncRNA FOXD2-AS1 in gallbladder cancer and t...

journal_title：Gene therapy

authors： Gao J,Dai C,Yu X,Yin XB,Liao WJ,Huang Y,Zhou F

更新日期：2020-09-11 00:00:00

abstract：:Adeno-associated virus (AAV) derived vectors are considered highly eligible vehicles for human gene therapy. Not only do they possess many great potential for clinical applications due to their wide range of tissue targets but also their excellent preclinical safety profile makes them particularly suitable candidates ...

journal_title：Gene therapy

authors： Le Bec C,Douar AM

更新日期：2006-05-01 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：:Current generation adenovirus (Ad) vectors are deleted for the E1 region of genes and require propagation in E1 expressing 293 cells. Expression of genes delivered by Ad vectors into immunocompetent hosts is generally transient since the current vectors are not completely replication defective. Viral proteins expresse...

journal_title：Gene therapy

authors： Amalfitano A,Chamberlain JS

更新日期：1997-03-01 00:00:00

abstract：:The use of adenoviral vectors as potent gene delivery systems requires expression of the Coxsackievirus/adenovirus receptor (CVADR) on the target cell surface. This receptor is important for virus attachment to the cell surface. For effective internalization of the vector into the target cell the integrins alpha(v)bet...

journal_title：Gene therapy

authors： Loskog A,Hedlund T,Wester K,de la Torre M,Philipson L,Malmström PU,Tötterman TH

更新日期：2002-05-01 00:00:00

abstract：:The pseudorabies virus (PRV) is a swine alpha herpes virus that is widely used as a neural tracer because of its marked neurotropism and transneuronal transmissibility (Card et al., 1991, 1992; Strack and Loewy 1990). PRV has been used to retrogradely identify spinal cord and brainstem connections to various periphera...

journal_title：Gene therapy

authors： Jasmin L,Tarczy-Hornoch K,Wang H,Levine JD,Basbaum AI

更新日期：1994-01-01 00:00:00

abstract：:Prenatal gene therapy has been considered for Herlitz junctional epidermolysis bullosa (H-JEB), a lethal genodermatosis caused by the absence of any of the three subunits of laminin-5, resulting from birth in widespread blistering and erosions of skin and mucosae. To investigate this strategy in an animal model, adeno...

journal_title：Gene therapy

authors： Mühle C,Neuner A,Park J,Pacho F,Jiang Q,Waddington SN,Schneider H

更新日期：2006-12-01 00:00:00

abstract：:A novel class of cationic hyperbranched polymers, containing branched oligoethylenimine (OEI 800 Da) as core, diacrylate esters as linkers and oligoamines as surface modification, was synthesized and evaluated regarding their structure-activity relationship as gene carriers. We show that pseudodendritic core character...

journal_title：Gene therapy

authors： Russ V,Elfberg H,Thoma C,Kloeckner J,Ogris M,Wagner E

更新日期：2008-01-01 00:00:00

abstract：:Selection of targeted vectors from virus display peptide libraries is a versatile and efficient approach to improve vector specificity and efficiency. This strategy has been used to target various cell types in vitro. Here, we report the screening of an adeno-associated virus type 2 (AAV2) display peptide library in v...

journal_title：Gene therapy

authors： Ying Y,Müller OJ,Goehringer C,Leuchs B,Trepel M,Katus HA,Kleinschmidt JA

更新日期：2010-08-01 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：:Neonatal AAV8-mediated Factor IX (F.IX) gene delivery was applied as a model for exploring mechanisms of tolerance induction during immune ontogeny. Intraperitoneal delivery of AAV8/ Factor IX (hF.IX) during weeks 1-4 of life, over a 20-fold dose range, directed stable hF.IX expression, correction of coagulopathy in F...

journal_title：Gene therapy

authors： Shi Y,Falahati R,Zhang J,Flebbe-Rehwaldt L,Gaensler KM

更新日期：2013-10-01 00:00:00

abstract：:Human adipose tissue mesenchymal stromal cells (AMSCs) share common traits, including similar differentiation potential and cell surface markers, with their bone marrow counterparts. Owing to their general availability, higher abundance and ease of isolation AMSCs may be convenient autologous delivery vehicles for loc...

journal_title：Gene therapy

authors： Vilalta M,Dégano IR,Bagó J,Aguilar E,Gambhir SS,Rubio N,Blanco J

更新日期：2009-04-01 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：:Active disease of tuberculosis (TB) can be developed decades later by either a relapse of the initial infection (endogenous reactivation) or by an entrance of the secondary infection (exogenous reinfection), since the current chemotherapy cannot lead to complete elimination of tuberculosis. Although the immunotherapeu...

journal_title：Gene therapy

authors： Ha SJ,Jeon BY,Youn JI,Kim SC,Cho SN,Sung YC

更新日期：2005-04-01 00:00:00

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：:Breast cancer is the leading cancer diagnosed in women and the second leading cause of cancer-related deaths in women. Current limitations to standard chemotherapy in the clinic are extensively researched, including problems arising from repeated treatments with the same drugs. The phenomenon that cancer cells become ...

journal_title：Gene therapy

authors： Jones SK,Merkel OM

更新日期：2016-12-01 00:00:00

abstract：:Intramuscular (i.m.) injection of a plasmid encoding human carcinoembryonic antigen (CEA) elicited immunity against transplanted syngeneic (C57BL/6) CEA-positive Lewis lung carcinoma (CEA/LLC) cells, but tumors still appeared in all mice. In wild-type mice, coinjection of an IL-12 plasmid markedly enhanced anti-CEA hu...

journal_title：Gene therapy

authors： Song K,Chang Y,Prud'homme GJ

更新日期：2000-09-01 00:00:00

abstract：:Chimeric antigen receptors (CARs, immunoreceptors) are frequently used to redirect T cells with pre-defined specificity, in particular towards tumour cells for use in adoptive immunotherapy of malignant diseases. Specific targeting is mediated by an extracellularly located antibody-derived binding domain, which is joi...

journal_title：Gene therapy

authors： Hombach A,Hombach AA,Abken H

更新日期：2010-10-01 00:00:00

abstract：:Adeno-associated virus (AAV)-mediated gene delivery systems have been shown to be effective tools for gene manipulation in the inner ear. For example, hair cells (HCs) and multiple other cell types can be transduced by the local injection of AAVs into the inner ear. However, application of the AAV-mediated CRISPR/Cas9...

journal_title：Gene therapy

authors： Kang W,Zhao X,Sun Z,Dong T,Jin C,Tong L,Zhu W,Tao Y,Wu H

更新日期：2020-08-01 00:00:00

abstract：:Urea cycle defects presenting in the neonatal period with hyperammonaemia are associated with high morbidity and mortality, and necessitate liver transplantation for long-term management. Gene therapy is therefore an attractive possibility, with vectors based on adeno-associated virus (rAAV) currently showing exciting...

journal_title：Gene therapy

authors： Cunningham SC,Kok CY,Spinoulas A,Carpenter KH,Alexander IE

更新日期：2013-12-01 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：:Recently developed helper virus-free methods of herpes simplex virus (HSV) amplicon vector packaging provide stocks that are virtually devoid of the cytotoxic component normally associated with traditional helper virus-based packaging methods. These approaches involve cotransfection of amplicon plasmid DNA with either...

journal_title：Gene therapy

authors： Bowers WJ,Howard DF,Brooks AI,Halterman MW,Federoff HJ

更新日期：2001-01-01 00:00:00

abstract：:We introduce a lung inflation-fixation protocol to examine the distribution and gene transfer efficiency of fluorescently tagged lipoplexes using fluorescence confocal microscopy within thick lung tissue sections. Using this technique, we tested the hypothesis that factors related to lipoplex distribution were the pre...

journal_title：Gene therapy

authors： Uyechi LS,Gagné L,Thurston G,Szoka FC Jr

更新日期：2001-06-01 00:00:00