Intercellular trafficking and enhanced in vivo antitumour activity of a non-virally delivered P27-VP22 fusion protein.

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：:This study was designed to determine whether Coxsackie adenovirus receptor (CAR) and alpha nu beta3/alpha nu beta5 integrin co-receptors are involved in adenovirus gene transfer in the rat cochlea. We find that CAR and integrin co-receptors are expressed in every cell subtype transduced by the adenoviral vector Ad5 De...

journal_title：Gene therapy

authors： Venail F,Wang J,Ruel J,Ballana E,Rebillard G,Eybalin M,Arbones M,Bosch A,Puel JL

更新日期：2007-01-01 00:00:00

abstract：:A persistent obstacle in the use of vector systems for gene therapy has been the inability to attain high-level expression of the target gene in primary cells in vivo. The MFG retroviral vector was designed to yield improved expression over the widely used N2 or LN vectors; however, the molecular basis for this effect...

journal_title：Gene therapy

authors： Krall WJ,Skelton DC,Yu XJ,Riviere I,Lehn P,Mulligan RC,Kohn DB

更新日期：1996-01-01 00:00:00

abstract：:This work demonstrates that electrical muscle stimulation markedly increases the transfection efficiency of an intramuscular injection of plasmid DNA. In soleus or extensor digitorum longus muscles of adult rats the percentage of transfected fibers increased from about 1 to more than 10. The number of transfected fibe...

journal_title：Gene therapy

authors： Mathiesen I

更新日期：1999-04-01 00:00:00

abstract：:Hydrodynamic gene delivery to the liver is an attractive approach for clinical liver gene therapy, but critical aspects of technique remain uncertain. There has not been to date any report of high levels of hydrodynamic gene delivery to the liver, except in rodents. Regional hydrodynamic delivery to individual lobes/s...

journal_title：Gene therapy

authors： Sawyer GJ,Zhang X,Fabre JW

更新日期：2010-04-01 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：:Retroviral vectors have become the primary tool for gene delivery into hematopoietic cells, including T lymphocytes. Lentiviral vectors offer an advantage over Moloney murine leukemia virus (MuLV) vectors because of their ability to translocate across an intact nuclear membrane and integrate into the genome of nonprol...

journal_title：Gene therapy

authors： Dardalhon V,Herpers B,Noraz N,Pflumio F,Guetard D,Leveau C,Dubart-Kupperschmitt A,Charneau P,Taylor N

更新日期：2001-02-01 00:00:00

abstract：:The current therapies to treat hepatitis B virus (HBV) infection are limited. Recently, clustered regularly interspaced short palindromic repeat (CRISPR) systems, originally identified in bacteria and archaea, have been found to consist of an RNA-based adaptive immune system that degrades complimentary sequences of in...

journal_title：Gene therapy

authors： Zhen S,Hua L,Liu YH,Gao LC,Fu J,Wan DY,Dong LH,Song HF,Gao X

更新日期：2015-05-01 00:00:00

abstract：:Adenovirus vectors have great potential in cancer gene therapy. Targeting of cancer-testis (CT) antigens, which are specifically presented at the surface of tumor cells by human leukocyte antigen (HLA) class I molecules, is an attractive option. In this study, a single-chain T-cell receptor (scTCR) directed against th...

journal_title：Gene therapy

authors： de Vrij J,Uil TG,van den Hengel SK,Cramer SJ,Koppers-Lalic D,Verweij MC,Wiertz EJ,Vellinga J,Willemsen RA,Hoeben RC

更新日期：2008-07-01 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：:Plasma apolipoprotein AI (apoAI) and lecithin-cholesterol acyltransferase (LCAT) play important roles in reverse cholesterol transport, promoting the removal of excess cholesterol from peripheral cells and reducing formation of atherosclerotic lesions. Gene augmentation of either apoAI or LCAT, or both, are thus attra...

journal_title：Gene therapy

authors： Fan L,Drew J,Dunckley MG,Owen JS,Dickson G

更新日期：1998-10-01 00:00:00

abstract：:Gene therapy is a potential new strategy for the treatment of cardiovascular disease. The most efficacious method of gene delivery remains a key hurdle to effective gene therapy. We present the application of a novel, nonviral gene delivery system (TerplexDNA) to augment myocardial transfection. The hearts of New Zeal...

journal_title：Gene therapy

authors： Affleck DG,Yu L,Bull DA,Bailey SH,Kim SW

更新日期：2001-03-01 00:00:00

abstract：:Adeno-associated virus (AAV) gene therapy constitutes a powerful tool for the treatment of neurodegenerative diseases. While AAVs are generally administered systemically to newborns in preclinical studies of neurological disorders, in adults the maturity of the blood-brain barrier (BBB) must be considered when selecti...

journal_title：Gene therapy

authors： Bey K,Ciron C,Dubreil L,Deniaud J,Ledevin M,Cristini J,Blouin V,Aubourg P,Colle MA

更新日期：2017-05-01 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：:Advances in islet transplantation have encouraged efforts to create alternative insulin-secreting cells that overcome limitations associated with current therapies. We have recently demonstrated durable correction of murine and porcine diabetes by syngeneic and autologous implantation, respectively, of primary hepatoc...

journal_title：Gene therapy

authors： Chen NK,Tan SY,Udolph G,Kon OL

更新日期：2010-05-01 00:00:00

abstract：:Human adenovirus (AV) is a favored vector for delivery of therapeutic genes into certain target cells, such as skeletal muscle cells for gene therapy. Here we show that replication-defective (E1 + E3 deleted) human type 5 adenovirus (AV) recombinants containing a reporter gene insert (RSV-luciferase or RSV-Lux) can ve...

journal_title：Gene therapy

authors： Acsadi G,Jani A,Huard J,Blaschuk K,Massie B,Holland P,Lochmüller H,Karpati G

更新日期：1994-09-01 00:00:00

abstract：:Citrullinemia type 1 (CTLN1) is an autosomal recessive disorder of metabolism caused by a deficiency of argininosuccinate synthetase. Despite optimal management, CTLN1 patients still suffer from lethal metabolic instability and experience life-threatening episodes of acute hyperammonemia. A murine model of CTLN1 (fold...

journal_title：Gene therapy

authors： Chandler RJ,Tarasenko TN,Cusmano-Ozog K,Sun Q,Sutton VR,Venditti CP,McGuire PJ

更新日期：2013-12-01 00:00:00

abstract：:Defects of the mitochondrial genome cause a wide variety of clinical disorders. Except for rare cases where surgery or transplant is indicated, there is no effective treatment for patients. Genetic-based therapies are consequently being considered. On account of the difficulties associated with mitochondrial (mt) tran...

journal_title：Gene therapy

authors： Kyriakouli DS,Boesch P,Taylor RW,Lightowlers RN

更新日期：2008-07-01 00:00:00

abstract：:Virus-directed enzyme prodrug therapy utilizing the bacterial enzyme nitroreductase delivered by a replication-defective adenovirus vector to activate the prodrug CB1954 is a promising strategy currently undergoing clinical trials in patients with a range of cancers. Similarly, selectively replicating oncolytic adenov...

journal_title：Gene therapy

authors： Palmer DH,Chen MJ,Searle PF,Kerr DJ,Young LS

更新日期：2005-08-01 00:00:00

abstract：:In high-risk patients, the ideal cardiovascular gene therapy requires a strategy that provides long-term protection of myocardium against episodes of ischemic/reperfusion injury. We report the development of an efficient, long-lasting pre-emptive gene therapy strategy in a rat model of ischemic-reperfusion (I/R) injur...

journal_title：Gene therapy

authors： Agrawal RS,Muangman S,Layne MD,Melo L,Perrella MA,Lee RT,Zhang L,Lopez-Ilasaca M,Dzau VJ

更新日期：2004-06-01 00:00:00

abstract：:Intracardiac gene transfer and gene therapy have been investigated with different vector systems. Here we used adeno-associated virus (AAV) vectors to deliver either a reporter gene or a therapeutic gene into the heart of golden Syrian hamsters. The method of gene delivery was direct infusion of the AAV2 vectors into ...

journal_title：Gene therapy

authors： Li J,Wang D,Qian S,Chen Z,Zhu T,Xiao X

更新日期：2003-10-01 00:00:00

abstract：:The development of efficient systems for in vivo gene transfer to the central nervous system (CNS) may provide a useful therapeutic strategy for the alleviation of several neurological disorders. In this study, we evaluated the feasibility of nonviral gene therapy to the CNS mediated by cationic liposomes. We present ...

journal_title：Gene therapy

authors： da Cruz MT,Cardoso AL,de Almeida LP,Simões S,de Lima MC

更新日期：2005-08-01 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：:To confer adenovirus vectors (AdV), the feature of integration into the host cell genome hybrid vectors were characterized in vitro, which express vectors derived from the prototypic foamy virus (FV) in the backbone of a high-capacity AdV. FVs constitute a subfamily of retroviruses with a distinct replication pathway ...

journal_title：Gene therapy

authors： Picard-Maureau M,Kreppel F,Lindemann D,Juretzek T,Herchenröder O,Rethwilm A,Kochanek S,Heinkelein M

更新日期：2004-04-01 00:00:00

abstract：:For certain gene therapy applications, the simultaneous delivery of multiple genes would allow for novel therapies. In the case of adeno-associated virus (AAV) vectors, the limited packaging capacity greatly restricts current methods of carrying multiple transgene cassettes. To address this issue, a recombinant AAV (r...

journal_title：Gene therapy

authors： Foti SB,Samulski RJ,McCown TJ

更新日期：2009-11-01 00:00:00

abstract：:The role of gap junctional intercellular communication (GJIC) in bystander killing with herpes simplex virus thymidine kinase (HSV-TK) and ganciclovir (GCV) was evaluated in U251 cells expressing a dominant-negative connexin 43 cDNA (DN14), and in HeLa cells, reportedly devoid of connexin protein. These cell lines bot...

journal_title：Gene therapy

authors： Gentry BG,Im M,Boucher PD,Ruch RJ,Shewach DS

更新日期：2005-07-01 00:00:00

abstract：:We previously demonstrated that recombinant adeno-associated virus vectors based on serotype 2 (rAAV2) can direct transgene expression in salivary gland cells in vitro and in vivo. However, it is not known how other rAAV serotypes perform when infused into salivary glands. The capsids of serotypes 4 and 5 are distinct...

journal_title：Gene therapy

authors： Katano H,Kok MR,Cotrim AP,Yamano S,Schmidt M,Afione S,Baum BJ,Chiorini JA

更新日期：2006-04-01 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：: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：:Recombinant adeno-associated virus type 2 (rAAV) vectors have recently been used to achieve long-term, high level transduction in vivo. Further development of rAAV vectors for clinical use requires significant technological improvements in large-scale vector production. In order to facilitate the production of rAAV ve...

journal_title：Gene therapy

authors： Conway JE,Rhys CM,Zolotukhin I,Zolotukhin S,Muzyczka N,Hayward GS,Byrne BJ

更新日期：1999-06-01 00:00:00