Intrabodies: turning the humoral immune system outside in for intracellular immunization.

abstract：:The potential for gene therapy to be an effective treatment for cystic fibrosis has been hampered by the limited gene transfer efficiency of current vectors. We have shown that recombinant Sendai virus (SeV) is highly efficient in mediating gene transfer to differentiated airway epithelial cells, because of its capaci...

journal_title：Gene therapy

authors： Ferrari S,Griesenbach U,Iida A,Farley R,Wright AM,Zhu J,Munkonge FM,Smith SN,You J,Ban H,Inoue M,Chan M,Singh C,Verdon B,Argent BE,Wainwright B,Jeffery PK,Geddes DM,Porteous DJ,Hyde SC,Gray MA,Hasegawa M,Alton

更新日期：2007-10-01 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：: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 oncotropic phenotypes of several viruses correlate with tumor-associated deficiencies within interferon (IFN) signaling pathways. This observation formed the conceptual basis for developing oncolytic viruses deleted for viral proteins that inhibit the host IFN-dependent antiviral response, such as herpes simplex v...

journal_title：Gene therapy

authors： Sobol PT,Hummel JL,Rodrigues RM,Mossman KL

更新日期：2009-09-01 00:00:00

abstract：:The efficient delivery of genetic material to the developing fetal brain represents a powerful research tool and a means to supply therapy in a number of neonatal lethal neurological disorders. In this study, we have delivered vectors based upon adenovirus serotype 5 (Ad5) and adeno-associated virus (AAV) pseudotypes ...

journal_title：Gene therapy

authors： Rahim AA,Wong AM,Ahmadi S,Hoefer K,Buckley SM,Hughes DA,Nathwani AN,Baker AH,McVey JH,Cooper JD,Waddington SN

更新日期：2012-09-01 00:00:00

abstract：:Attempts have been made to use various forms of cellular vectors to deliver therapeutic genes to diseased tissues like malignant tumours. However, this approach has proved problematic due to the poor uptake of these vectors by the target tissue. We have devised a novel way of using magnetic nanoparticles (MNPs) to enh...

journal_title：Gene therapy

authors： Muthana M,Scott SD,Farrow N,Morrow F,Murdoch C,Grubb S,Brown N,Dobson J,Lewis CE

更新日期：2008-06-01 00:00:00

abstract：:Herpes simplex virus type 1 (HSV-1) is a neurotrophic human pathogen that naturally persists in neurons in a latent state and carries a large number of viral functions which can be replaced by foreign genes to create a vector for gene therapy applications. In this report we describe a two-step method for insertion/del...

journal_title：Gene therapy

authors： Krisky DM,Marconi PC,Oligino T,Rouse RJ,Fink DJ,Glorioso JC

更新日期：1997-10-01 00:00:00

abstract：:Genetic engineering of T cells for adoptive immunotherapy in cancer patients has shown significant promise. To ensure optimal antitumor activity and safety, the simultaneous expression of multiple genes is frequently required, and short viral-derived 2A sequences are increasingly preferred for this purpose. Concerns e...

journal_title：Gene therapy

authors： Arber C,Abhyankar H,Heslop HE,Brenner MK,Liu H,Dotti G,Savoldo B

更新日期：2013-09-01 00:00:00

abstract：:Intervertebral disc degeneration (IVDD) is a multi-factorial process characterized by phenotypic and genotypic changes, which leads to low back pain and disability. Prolonged imbalance between anabolism and catabolism in discs alters their composition resulting in progressive loss of proteoglycans and hydration leadin...

journal_title：Gene therapy

authors： Sampara P,Banala RR,Vemuri SK,Av GR,Gpv S

更新日期：2018-04-01 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

abstract：:Recombinant Semliki Forest virus (SFV) is an attractive viral vector system owing to its ability to allow high efficiency of viral protein expression. To produce recombinant pseudotyped human immunodeficiency virus type 1 (HIV-1) virions, we designed a chimeric SFV/HIV vector system that contains both the HIV-1 cis- a...

journal_title：Gene therapy

authors： Del Vecchio C,Calistri A,Lombardi G,Celegato M,Biasolo MA,Palù G,Parolin C

更新日期：2009-02-01 00:00:00

abstract：:To achieve high transgene expression in the liver, we have compared the reporter gene expression among various murine retroviral long terminal repeats (LTRs) or leader sequences in vitro. Transient reporter gene expression assays revealed the highest gene expression by the polycythemic strain of spleen focus-forming v...

journal_title：Gene therapy

authors： Ohnishi N,Itoh K,Itoh Y,Baum C,Higashitsuji H,Yamaguchi K,Tsuji T,Okanoue T,Fujita J

更新日期：2002-02-01 00:00:00

abstract：:Chemically inducible gene switches that regulate expression of endogenous genes have multiple applications for basic gene expression research and gene therapy. Single-chain zinc-finger transcription factors that utilize either estrogen receptor homodimers or retinoid X receptor-alpha/ecdysone receptor heterodimers are...

journal_title：Gene therapy

authors： Magnenat L,Schwimmer LJ,Barbas CF 3rd

更新日期：2008-09-01 00:00:00

abstract：:Prostate cancer is the most common cancer in men of the western world. To date, no effective treatment exists for metastatic prostate cancer and consequently, there is an urgent need to develop new and improved therapeutics. In recent years, the therapeutic potential of RNA interference (RNAi) has been extensively exp...

journal_title：Gene therapy

authors： Evans JC,McCarthy J,Torres-Fuentes C,Cryan JF,Ogier J,Darcy R,Watson RW,O'Driscoll CM

更新日期：2015-10-01 00:00:00

abstract：:Introduction of the herpes simplex virus type 1 thymidine kinase gene into tumor cells, followed by the administration of the antiherpes nucleoside analogue ganciclovir has been demonstrated to be effective in eliminating solid tumors in animals. The success of this combination treatment largely depends on the bystand...

journal_title：Gene therapy

authors： Degrève B,De Clercq E,Balzarini J

更新日期：1999-02-01 00:00:00

abstract：:In-stent restenosis results exclusively from neointimal hyperplasia due to mechanical injury and a foreign body response to the prosthesis. Inflammation mediated by monocyte chemoattractant protein-1 (MCP-1) might therefore underlie in-stent restenosis. We recently devised a new strategy for anti-MCP-1 gene therapy by...

journal_title：Gene therapy

authors： Ohtani K,Usui M,Nakano K,Kohjimoto Y,Kitajima S,Hirouchi Y,Li XH,Kitamoto S,Takeshita A,Egashira K

更新日期：2004-08-01 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：:Newcastle disease virus (NDV) is an oncolytic paramyxovirus with a nonsegmented single-stranded RNA genome. In this report, a recombinant oncolytic NDV was passaged in human tumor xenografts and reisolated and characterized after two rounds of bioselection. Several isolates could be recovered that differed from the pa...

journal_title：Gene therapy

authors： Beier R,Hermiston T,Mumberg D

更新日期：2013-01-01 00:00:00

abstract：:Complexes of DNA and cationic lipid offer potential advantages for gene transfer to airway epithelia. However, we found that application of DNA-lipid (DMRIE-DOPE) complexes to primary cultures of human ciliated airway epithelia or explants of rabbit trachea generated only low levels of gene transfer. In contrast, when...

journal_title：Gene therapy

authors： Fasbender A,Zabner J,Zeiher BG,Welsh MJ

更新日期：1997-11-01 00:00:00

abstract：:Reactive oxygen species (ROS) have been implicated in the pathogenesis of rheumatoid arthritis (RA), while antioxidant enzymes, such as extracellular superoxide dismutase (EC-SOD) and catalase, block radical-induced events. The present study tested if the ex vivo transfer of EC-SOD and catalase genes alone or in combi...

journal_title：Gene therapy

authors： Dai L,Claxson A,Marklund SL,Feakins R,Yousaf N,Chernajovsky Y,Winyard PG

更新日期：2003-04-01 00:00:00

abstract：:Gyrate atrophy (GA) of the choroid and retina is an autosomal recessive chorioretinal degeneration, caused by deficiency of the mitochondrial matrix enzyme ornithine-delta-aminotransferase (OAT). This deficiency results in the accumulation of ornithine in the body fluids and leads to hyperornithinemia. Although the cl...

journal_title：Gene therapy

authors： Lacorazza HD,Jendoubi M

更新日期：1995-01-01 00:00:00

abstract：:Hepatic fibrosis is a common outcome of chronic liver diseases. In schistosomiasis, chronic parasite egg-induced granuloma formation can lead to fibrosis, which is immunologically characterized by the dominant Th2 response. Recently, it has been shown that gene therapy is an attractive approach for the treatment of he...

journal_title：Gene therapy

authors： Zhang LH,Pan JP,Yao HP,Sun WJ,Xia DJ,Wang QQ,He L,Wang J,Cao X

更新日期：2001-09-01 00:00:00

abstract：:In vitro culture systems of human myogenic cells contribute greatly to elucidation of the molecular mechanisms underlying terminal myogenic differentiation and symptoms of neuromuscular diseases. However, human myogenic cells have limited ability to proliferate in culture. We have established an improved immortalizati...

journal_title：Gene therapy

authors： Shiomi K,Kiyono T,Okamura K,Uezumi M,Goto Y,Yasumoto S,Shimizu S,Hashimoto N

更新日期：2011-09-01 00:00:00

abstract：:Vectors conjugated with ligands recognized by cell surface receptors are of interest for cystic fibrosis gene therapy since these vectors would allow cell-specific targeting. However, an efficient and specific uptake may be abrogated by a subsequent intracellular trafficking leading to an inefficient gene transfer. Th...

journal_title：Gene therapy

authors： Fajac I,Grosse S,Briand P,Monsigny M

更新日期：2002-06-01 00:00:00

abstract：:Bone marrow stromal cells (MSCs) are pluripotent cells capable of differentiation into several tissue types. This present study was performed to determine their functional neoangiogenic potential in vivo. Whole bone marrow was harvested from C57Bl/6 mice, and the adherent cellular fraction was culture expanded for 14 ...

journal_title：Gene therapy

authors： Al-Khaldi A,Eliopoulos N,Martineau D,Lejeune L,Lachapelle K,Galipeau J

更新日期：2003-04-01 00:00:00

abstract：:Conditionally replicating adenoviruses (CRAd) are a promising class of gene therapy agents that can overcome already known glioblastoma (GBM) resistance mechanisms but have limited distribution upon direct intratumoral (i.t.) injection. Collagen bundles in the extracellular matrix (ECM) have an important role in inhib...

journal_title：Gene therapy

authors： Thaci B,Ulasov IV,Ahmed AU,Ferguson SD,Han Y,Lesniak MS

更新日期：2013-03-01 00:00:00

abstract：:Newly isolated serotypes of AAV readily cross the endothelial barrier to provide efficient transgene delivery throughout the body. However, tissue-specific expression is preferred in most experimental studies and gene therapy protocols. Previous efforts to restrict gene expression to the myocardium often relied on dir...

journal_title：Gene therapy

authors： Prasad KM,Xu Y,Yang Z,Acton ST,French BA

更新日期：2011-01-01 00:00:00

abstract：:The bystander effect (BSE) is an interesting and important property of the herpes thymidine kinase/ganciclovir (hTK/GCV) system of gene therapy for cancer. With the BSE, not only are the hTK expressing cells killed upon ganciclovir (GCV) exposure but also neighboring wild-type tumor cells. On testing a large number of...

journal_title：Gene therapy

authors： Touraine RL,Ishii-Morita H,Ramsey WJ,Blaese RM

更新日期：1998-12-01 00:00:00

abstract：:Recombinant Autographa californica multiple nuclear polyhedrosis viruses (AcMNPV) have recently been shown to transduce mammalian cells in vitro. Since baculoviruses offer many advantages over viruses currently used in gene therapy, we have tested them for in vivo gene transfer by constructing a baculovirus bearing a ...

journal_title：Gene therapy

authors： Airenne KJ,Hiltunen MO,Turunen MP,Turunen AM,Laitinen OH,Kulomaa MS,Ylä-Herttuala S

更新日期：2000-09-01 00:00:00

abstract：:Both replication-incompetent and replication-selective adenoviruses are being developed for the treatment of cancer and other diseases. Concerns have been raised about the safety of intra-vascular adenovirus administration following a patient death on a clinical trial with a replication-defective adenovirus. In additi...

journal_title：Gene therapy

authors： Reid T,Galanis E,Abbruzzese J,Sze D,Andrews J,Romel L,Hatfield M,Rubin J,Kirn D

更新日期：2001-11-01 00:00:00