A retroviral vector system 'STITCH' in combination with an optimized single chain antibody chimeric receptor gene structure allows efficient gene transduction and expression in human T lymphocytes.

abstract：:Constitutive expression of short hairpin RNAs (shRNAs) may cause cellular toxicity in vivo and using microRNA (miRNA) scaffolds can circumvent this problem. Previously, we have shown that embedding small interfering RNA sequences targeting apolipoprotein B100 (ApoB) in shRNA (shApoB) or miRNA (miApoB) scaffolds result...

journal_title：Gene therapy

authors： Maczuga P,Verheij J,van der Loos C,van Logtenstein R,Hooijer G,Martier R,Borel F,Lubelski J,Koornneef A,Blits B,van Deventer S,Petry H,Konstantinova P

更新日期：2014-01-01 00:00:00

abstract：:The concept and application of ethical principles in the context of medical research is changing rapidly. This is especially true for fast moving fields such as gene therapy, a relatively new but rapidly maturing field offering opportunities to influence health at a fundamental level, which may become a cornerstone of...

journal_title：Gene therapy

authors： Spink J,Geddes D

更新日期：2004-11-01 00:00:00

abstract：:Viral gene vectors often rely on packaging cell lines, which provide the necessary factors in trans for the formation of virus-like particles. Previously, we reported on a first-generation packaging cell line for gene vectors, which are based on the B-lymphotropic Epstein-Barr virus (EBV), a human gamma-herpesvirus. T...

journal_title：Gene therapy

authors： Hettich E,Janz A,Zeidler R,Pich D,Hellebrand E,Weissflog B,Moosmann A,Hammerschmidt W

更新日期：2006-05-01 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：:Viral therapy of cancer includes strategies such as viral transduction of tumour cells with 'suicide genes', using viral infection to trigger immune-mediated tumour cell death and using oncolytic viruses for their direct anti-tumour action. However, problems still remain in terms of adequate viral delivery to tumours....

journal_title：Gene therapy

authors： Pencavel T,Seth R,Hayes A,Melcher A,Pandha H,Vile R,Harrington KJ

更新日期：2010-08-01 00:00:00

abstract：:Oligonucleotides (ODNs) show great promise in their ability to specifically inhibit single gene expression but must cross the cell membrane, escape the endosomal vesicle, and possibly traverse the nuclear membrane to arrive at their intracellular target molecules. In an attempt to improve the delivery of phosphodieste...

journal_title：Gene therapy

authors： Ebbinghaus SW,Vigneswaran N,Miller CR,Chee-Awai RA,Mayfield CA,Curiel DT,Miller DM

更新日期：1996-04-01 00:00:00

abstract：:Replication defective vectors derived from simple retroviruses or the more complex genomes of lentiviruses continue to offer the advantages of long-term expression, cell and tissue specific tropism, and large packaging capacity for the delivery of therapeutic genes. The occurrence of adverse events caused by insertion...

journal_title：Gene therapy

authors： Sinn PL,Sauter SL,McCray PB Jr

更新日期：2005-07-01 00:00:00

abstract：:We introduce an injectable system for the formation of a biodegradable DNA-containing implant that releases DNA over a 2-month period to provide a robust and prolonged gene expression at the site. Sustained delivery of the appropriate plasmid DNA resulted in sustained expression of luciferase, the persistent appearanc...

journal_title：Gene therapy

authors： Eliaz RE,Szoka FC Jr

更新日期：2002-09-01 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：:A fully intact immune system would be expected to hinder the efficacy of oncolytic virotherapy by inhibiting viral replication. Simultaneously, however, it may also enhance antitumor therapy through initiation of proinflammatory, antiviral cytokine responses at the tumor site. The aim of this study was to investigate ...

journal_title：Gene therapy

authors： Galivo F,Diaz RM,Wongthida P,Thompson J,Kottke T,Barber G,Melcher A,Vile R

更新日期：2010-02-01 00:00:00

abstract：:In this study, we describe a simple system in which human keratinocytes can be redirected to an alternative differentiation pathway. We transiently transfected freshly isolated human skin keratinocytes with the single transcription factor OCT4. Within 2 days these cells displayed expression of endogenous embryonic gen...

journal_title：Gene therapy

authors： Racila D,Winter M,Said M,Tomanek-Chalkley A,Wiechert S,Eckert RL,Bickenbach JR

更新日期：2011-03-01 00:00:00

abstract：:Tissue-specific promoters for gene therapy are typically too big for adeno-associated virus (AAV) vectors; thus, the exploration of small effective non-viral regulatory elements is of particular interest. Wild-type AAV can specifically integrate into a region on human chromosome 19 termed AAVS1. Earlier work has deter...

journal_title：Gene therapy

authors： Li C,Hirsch M,Carter P,Asokan A,Zhou X,Wu Z,Samulski RJ

更新日期：2009-01-01 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：:The prognosis of pancreatic adenocarcinoma is poor and current treatment ineffective. A novel treatment strategy is described here using a mouse model system for pancreatic cancer. Cells that have been genetically modified to express the cytochrome P450 2B1 enzyme are encapsulated in cellulose sulphate and implanted i...

journal_title：Gene therapy

authors： Löhr M,Müller P,Karle P,Stange J,Mitzner S,Jesnowski R,Nizze H,Nebe B,Liebe S,Salmons B,Günzburg WH

更新日期：1998-08-01 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：: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：:Adenoviral vectors deleted of E1 are attractive vehicles for in vivo gene therapy because efficient gene transfer can be achieved. Immune responses to the vector and vector-transduced cells lead to destruction of target cells, inflammation and difficulties with vector readministration. Immune effectors have been ident...

journal_title：Gene therapy

authors： Jooss K,Turka LA,Wilson JM

更新日期：1998-03-01 00:00:00

abstract：:Because periodontal ligament (PDL) cells are reported to contain progenitor or stem cell populations, they are considered a beneficial cell source for clinical periodontal regeneration. Both bone morphogenetic protein 4 (BMP4) and human telomerase reverse transcriptase (hTERT) have essential roles in the modulation of...

journal_title：Gene therapy

authors： Mi HW,Lee MC,Fu E,Chow LP,Lin CP

更新日期：2011-05-01 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：:Green fluorescent protein (GFP) is a widely used intracellular reporter molecule to assess gene transfer and expression. A potential use for GFP is as a co-expressed marker, to select and enrich gene-modified cells by flow cytometry. Processed peptides derived from GFP and presented by the major histocompatibility com...

journal_title：Gene therapy

authors： Stripecke R,Carmen Villacres M,Skelton D,Satake N,Halene S,Kohn D

更新日期：1999-07-01 00:00:00

abstract：:We have constructed two recombinant adeno-associated virus (AAV) vectors (pJJ-3GC and pJJ-3ASA) which contained either the human glucocerebrosidase (GC) or arylsulfatase A (ASA) cDNA under the control of an SV40 promoter. These plasmids were co-transfected to 293 cells with a helper plasmid containing trans-acting AAV...

journal_title：Gene therapy

authors： Wei JF,Wei FS,Samulski RJ,Barranger JA

更新日期：1994-07-01 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：:Overexpression of vascular endothelial growth factor (VEGF) and its cognate receptor KDR has been linked to a more aggressive phenotype of human prostate carcinomas. The importance of signal transduction through the VEGF receptor 2 is illustrated by use of soluble KDR, which binds to VEGF and sequesters this ligand be...

journal_title：Gene therapy

authors： Kaliberov SA,Kaliberova LN,Buchsbaum DJ

更新日期：2005-03-01 00:00:00

abstract：:Intracerebral administration of recombinant adeno-associated vector (AAV) has been performed in several clinical trials. However, delivery into the brain requires multiple injections and is not efficient to target the spinal cord, thus limiting its applications. To assess widespread and less invasive strategies, we te...

journal_title：Gene therapy

authors： Hordeaux J,Dubreil L,Deniaud J,Iacobelli F,Moreau S,Ledevin M,Le Guiner C,Blouin V,Le Duff J,Mendes-Madeira A,Rolling F,Cherel Y,Moullier P,Colle MA

更新日期：2015-04-01 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：:Over the last years, RNA interference (RNAi) has become a widely used technique that permits the knock-down, and hence functional analysis, of individual genes in vertebrate cells. However, the high failure rate of the RNA molecules used in RNAi experiments continues to be a problem. In this paper, I describe a set of...

journal_title：Gene therapy

authors： Cullen BR

更新日期：2006-03-01 00:00:00

abstract：:Efficient transduction of the peripheral nervous system (PNS) is required for gene therapy of acquired and inherited neuropathies, neuromuscular diseases and for pain treatment. We have characterized the tropism and transduction efficiency of different adeno-associated vectors (AAV) pseudotypes after sciatic nerve inj...

journal_title：Gene therapy

authors： Homs J,Ariza L,Pagès G,Udina E,Navarro X,Chillón M,Bosch A

更新日期：2011-06-01 00:00:00

abstract：:This study uses a novel approach to gene therapy in which plasmid DNA is targeted to the pancreas in vivo using ultrasound-targeted microbubble destruction (UTMD) to achieve islet regeneration. Intravenous microbubbles carrying plasmids are destroyed within the pancreatic microcirculation by ultrasound, achieving loca...

journal_title：Gene therapy

authors： Chen S,Shimoda M,Wang MY,Ding J,Noguchi H,Matsumoto S,Grayburn PA

更新日期：2010-11-01 00:00:00

abstract：:Direct gene transfer for the treatment of human diseases requires a vector which can be administered efficiently, safely and repeatedly. Cationic liposomes represent one of the few examples that can meet these requirements. Currently, more than a dozen cationic liposome formulations have been reported. These liposomes...

journal_title：Gene therapy

authors： Gao X,Huang L

更新日期：1995-12-01 00:00:00

abstract：:Cytomegalovirus (CMV) promoter is often present in recombinant adenovirus vectors (AdVs) suitable for gene therapy, ensuring high levels of transgene production in a wide range of hosts. Despite this characteristic, the presence of the AdV genome in target cells and tissues typically lasts longer than transgene produc...

journal_title：Gene therapy

authors： Gaetano C,Catalano A,Palumbo R,Illi B,Orlando G,Ventoruzzo G,Serino F,Capogrossi MC

更新日期：2000-10-01 00:00:00