Intra-articular delivery of a herpes simplex virus IL-1Ra gene vector reduces inflammation in a rabbit model of arthritis.

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：:Intramuscular (i.m.) injection of plasmids followed by electropermeabilization is an efficient process to deliver genes into skeletal myofibers that permits proteins to be produced and secreted at therapeutically relevant levels. To further improve skeletal muscle as a bioreactor, we identified a formulation that elev...

journal_title：Gene therapy

authors： Nicol F,Wong M,MacLaughlin FC,Perrard J,Wilson E,Nordstrom JL,Smith LC

更新日期：2002-10-01 00:00:00

abstract：:We tested the longevity of gene expression provided by autonomously replicating vectors. The vectors contain segments of human genomic DNA that provide efficient replication initiation and sequences derived from Epstein-Barr virus that provide nuclear retention. In order to monitor gene expression, the vectors also ca...

journal_title：Gene therapy

authors： Wohlgemuth JG,Kang SH,Bulboaca GH,Nawotka KA,Calos MP

更新日期：1996-06-01 00:00:00

abstract：:Cationic lipid-DNA complexes (lipoplexes) have been widely used as gene transfer vectors which avoid the adverse immunogenicity and potential for viraemia of viral vectors. With the long-term aim of gene transfer into skeletal muscle in vivo, we describe a direct in vitro comparison of two commercially available catio...

journal_title：Gene therapy

authors： Dodds E,Dunckley MG,Naujoks K,Michaelis U,Dickson G

更新日期：1998-04-01 00:00:00

abstract：:Polyethylenimine (PEI) has been studied as an efficient nonviral gene transfer vector. Here, we report the biodistribution fates and safety of plasmid DNA intravenously administered in PEI complexes. Using pCMVbeta as a model gene, the biodistribution of plasmid DNA was measured by quantitative polymerase chain reacti...

journal_title：Gene therapy

authors： Oh YK,Kim JP,Yoon H,Kim JM,Yang JS,Kim CK

更新日期：2001-10-01 00:00:00

abstract：:The broad host cell range and high expression levels of transgenes are features that have made alphaviruses attractive for gene expression studies and gene therapy applications. Particularly, Semliki Forest virus vectors have been applied for large-scale production of recombinant membrane proteins for drug screening p...

journal_title：Gene therapy

authors： Lundstrom K

更新日期：2005-10-01 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：:The structure of 'stabilized plasmid-lipid particles' (SPLP) and their properties as systemic gene therapy vectors has been investigated. We show that SPLP can be visualized employing cryo-electron microscopy to be homogeneous particles of diameter 72 +/- 5 nm consisting of a lipid bilayer surrounding a core of plasmi...

journal_title：Gene therapy

authors： Tam P,Monck M,Lee D,Ludkovski O,Leng EC,Clow K,Stark H,Scherrer P,Graham RW,Cullis PR

更新日期：2000-11-01 00:00:00

abstract：:With the identification of stem cell plasticity several years ago, multiple reports raised hopes that tissue repair by stem cell transplantation could be within reach in the near future. Krause et al reported that a single purified hematopoietic stem cell not only repopulated the bone marrow of a host animal, but also...

journal_title：Gene therapy

authors： Kashofer K,Bonnet D

更新日期：2005-08-01 00:00:00

abstract：:betaTC-tet cells are conditionally immortalized pancreatic beta cells which can confer long-term correction of hyperglycemia when transplanted in syngeneic streptozocin diabetic mice. The use of these cells for control of type I diabetes in humans will require their encapsulation and transplantation in non-native site...

journal_title：Gene therapy

authors： Dupraz P,Rinsch C,Pralong WF,Rolland E,Zufferey R,Trono D,Thorens B

更新日期：1999-06-01 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：:A novel synthetic polypeptide designed as a DNA binding-molecule for liver-specific, receptor-mediated, gene transfer was used to selectively introduce reporter genes into liver cells in the form of plasmid DNA-ligand complexes. The polypeptide was a D-lysine/D-serine copolymer (Lys/Ser = 33/36 or 53/60) modified with...

journal_title：Gene therapy

authors： Hisayasu S,Miyauchi M,Akiyama K,Gotoh T,Satoh S,Shimada T

更新日期：1999-04-01 00:00:00

abstract：:Transplantation of allogeneic pancreatic islets is an effective approach to treat type 1 diabetes. To bypass the need for systemic administration of immunosuppression drugs following transplantation, approaches to genetically modify allogeneic islets to express anti-inflammatory, immunosuppressive, or antiapoptotic pr...

journal_title：Gene therapy

authors： Rehman KK,Wang Z,Bottino R,Balamurugan AN,Trucco M,Li J,Xiao X,Robbins PD

更新日期：2005-09-01 00:00:00

abstract：:Relaxin-3 is a neuropeptide that is abundantly expressed by discrete brainstem neuron populations that broadly innervate forebrain areas rich in the relaxin-3 G-protein-coupled-receptor, RXFP3. Acute and subchronic central administration of synthetic relaxin-3 or an RXFP3-selective agonist peptide, R3/I5, increase fee...

journal_title：Gene therapy

authors： Ganella DE,Callander GE,Ma S,Bye CR,Gundlach AL,Bathgate RA

更新日期：2013-07-01 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：:As soon as RNA interference (RNAi) was found to work in mammalian cells, research quickly focused on harnessing this powerful endogenous and specific mechanism of gene silencing for human therapy. RNAi uses small RNAs, less than 30 nucleotides in length, to suppress expression of genes with complementary sequences. Tw...

journal_title：Gene therapy

authors： Dykxhoorn DM,Palliser D,Lieberman J

更新日期：2006-03-01 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：:Replication-deficient retroviruses have been successfully utilized as vectors, offering an efficient, stable method of therapeutic gene delivery. Many examples exist proving this mode of integrative gene transfer is both effective and safe in cultured systems and clinical trials. Along with their success, severe side ...

journal_title：Gene therapy

authors： Papayannakos C,Daniel R

更新日期：2013-06-01 00:00:00

abstract：:We have shown that various forms of oligonucleotides, chimeric RNA-DNA oligonucleotide (RDO) and single-stranded oligodeoxynucleotide (ODN), are capable of chromosomal gene alterations in mammalian cells. Using two ODNs we corrected an inactivating mutation in the tyrosinase gene and introduced an activating mutation ...

journal_title：Gene therapy

authors： Alexeev V,Igoucheva O,Yoon K

更新日期：2002-12-01 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：:Studies reviewed show that lentiviral gene therapy directed either at inhibiting the synthesis of brain acetaldehyde generated from ethanol or at degrading brain acetaldehyde fully prevent ethanol intake by rats bred for their high alcohol preference. However, after animals have chronically consumed alcohol, the above...

journal_title：Gene therapy

authors： Yedy Israel,Quintanilla ME,Ezquer F,Morales P,Rivera-Meza M,Karahanian E,Ezquer M,Herrera-Marschitz M

更新日期：2019-11-01 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：: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：:Fibrodysplasia ossificans progressiva (FOP) is an autosomal dominant congenital disorder characterized by progressive heterotopic bone formation. Currently, no definitive treatment exists for FOP. The activin receptor type IA / activin-like kinase 2 (ACVR1/ALK2) gene has been identified as the responsible gene for FOP...

journal_title：Gene therapy

authors： Takahashi M,Katagiri T,Furuya H,Hohjoh H

更新日期：2012-07-01 00:00:00

abstract：:A replication-incompetent adenoviral vector encoding the heavy chain C-fragment (H(C)50) of botulinum neurotoxin type C (BoNT/C) was evaluated as a mucosal vaccine against botulism in a mouse model. Single intranasal inoculation of the adenoviral vector elicited a high level of H(C)50-specific IgG, IgG1 and IgG2a in s...

journal_title：Gene therapy

authors： Xu Q,Pichichero ME,Simpson LL,Elias M,Smith LA,Zeng M

更新日期：2009-03-01 00:00:00

abstract：:Delivery of a normal copy of CFTR cDNA to airway epithelia may provide a novel treatment for cystic fibrosis lung disease. Unfortunately, current vectors are inefficient because of limited binding to the apical surface of airway epithelia. We recently reported that incorporation of adenovirus in a calcium phosphate co...

journal_title：Gene therapy

authors： Walters R,Welsh M

更新日期：1999-11-01 00:00:00

abstract：:The role of two receptors, previously proposed to mediate the entry of adenoviruses into human cells, the coxsackie and adenovirus receptor (CAR) and the major histocompatibility complex (MHC) class I heavy chain has been investigated. The expression of MHC class I in many tumours is reduced or absent, therefore if th...

journal_title：Gene therapy

authors： McDonald D,Stockwin L,Matzow T,Blair Zajdel ME,Blair GE

更新日期：1999-09-01 00:00:00

abstract：:A stable single vial lipoplex formulation has been developed that can be stored frozen without losing either biological activity or physical stability. This formulation was identified by systematically controlling several formulation variables and without introducing either stabilizers or surfactants. Analytical assay...

journal_title：Gene therapy

authors： Zelphati O,Nguyen C,Ferrari M,Felgner J,Tsai Y,Felgner PL

更新日期：1998-09-01 00:00:00

abstract：:Insulin-like growth factor-I (IGF-I) and its receptor (IGF-IR) are essential for normal ocular development and are expressed in numerous ocular cell types including lens epithelial cells, retinal pigment epithelial cells, Müller cells and endothelial cells. Endothelial cell proliferation is a common feature of prolife...

journal_title：Gene therapy

authors： Shaw LC,Pan H,Afzal A,Calzi SL,Spoerri PE,Sullivan SM,Grant MB

更新日期：2006-05-01 00:00:00

abstract：:Short hairpin RNA (shRNA)-mediated gene regulation is a commonly used technique for gene manipulation. An efficient and safe delivery system is indispensable when shRNA is delivered into living organisms for gene therapy. Previous studies have proved that DNA and protein can be delivered into dendritic cells (DCs) by ...

journal_title：Gene therapy

authors： Zhang L,Zhang T,Wang L,Shao S,Chen Z,Zhang Z

更新日期：2014-07-01 00:00:00