Intravenous injection of oligodeoxynucleotides to the NF-kappaB binding site inhibits hepatic metastasis of M5076 reticulosarcoma in mice.

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：:Mesenchymal stem cells (MSC) are a group of clonogenic cells present among the bone marrow stroma and capable of multilineage differentiation into mesoderm-type cells such as osteoblasts, adipocytes and chondrocytes. Due to their ease of isolation and their differentiation potential, MSC are being introduced into clin...

journal_title：Gene therapy

authors： Abdallah BM,Kassem M

更新日期：2008-01-01 00:00:00

abstract：:We investigated the efficiency of activated polyamidoamine dendrimers, a new class of nonviral vectors, to transfect rabbit and human corneas in ex vivo culture. In addition to assessing the expression of a marker gene we have demonstrated that this approach can be used to induce the production of TNF receptor fusion ...

journal_title：Gene therapy

authors： Hudde T,Rayner SA,Comer RM,Weber M,Isaacs JD,Waldmann H,Larkin DF,George AJ

更新日期：1999-05-01 00:00:00

abstract：:The annual rate of kidney graft loss caused by chronic allograft nephropathy (CAN) has not improved over the past decade. Recent reports suggest that acute renal ischemia results in development of CAN. The goal of the present study was to assess the renoprotective potential and safety of hepatocyte growth factor (HGF)...

journal_title：Gene therapy

authors： Isaka Y,Yamada K,Takabatake Y,Mizui M,Miura-Tsujie M,Ichimaru N,Yazawa K,Utsugi R,Okuyama A,Hori M,Imai E,Takahara S

更新日期：2005-05-01 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：:Small interfering RNAs (siRNAs) are emerging as promising therapeutic tools. However, the widespread clinical application of such molecules as modulators of gene expression is still dependent on several aspects that limit their bioavailability. One of the most promising strategies to overcome the barriers faced by gen...

journal_title：Gene therapy

authors： Dharmapuri S,Peruzzi D,Marra E,Palombo F,Bett AJ,Bartz SR,Yong M,Ciliberto G,La Monica N,Buser CA,Toniatti C,Aurisicchio L

更新日期：2011-07-01 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：: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：:In 2012, we reported that 5 out of 11 subjects in a clinical trial (NCT00372320) administering AdhAQP1 to radiation-damaged parotid glands showed increased saliva flow rates and decreased symptoms over the initial 42 days. AdhAQP1 is a first-generation, E1-deleted, replication-defective, serotype 5 adenoviral vector e...

journal_title：Gene therapy

authors： Zheng C,Baum BJ,Liu X,Goldsmith CM,Perez P,Jang SI,Cotrim AP,McCullagh L,Ambudkar IS,Alevizos I

更新日期：2015-09-01 00:00:00

abstract：:The tumor-suppressor gene PTEN encodes a multifunctional phosphatase that is mutated in a variety of human cancers. PTEN inhibits the phosphatidylinositol 3-kinase pathway and downstream functions, including activation of Akt/protein kinase B (PKB), cell survival, and cell proliferation in tumor cells carrying mutant-...

journal_title：Gene therapy

authors： Saito Y,Swanson X,Mhashilkar AM,Oida Y,Schrock R,Branch CD,Chada S,Zumstein L,Ramesh R

更新日期：2003-11-01 00:00:00

abstract：:RNA interference (RNAi) is an evolutionarily conserved process by which plants and animals protect their genomes utilizing small, double-stranded RNAs to degrade target RNAs in a sequence-specific manner. Post-transcriptional gene silencing by these moieties can lead to degradation of both cellular and viral RNAs. It ...

journal_title：Gene therapy

authors： Zhou N,Fang J,Mukhtar M,Acheampong E,Pomerantz RJ

更新日期：2004-12-01 00:00:00

abstract：:Silencing of gene expression by small interfering RNAs (siRNAs) is rapidly becoming a powerful tool for genetic analysis of mammalian cells. The use of DNA-based plasmid vectors to achieve transient and stable expression of siRNA has been developed to avoid the problems of double-stranded oligonucleotides transfection...

journal_title：Gene therapy

authors： Sabbioni S,Callegari E,Manservigi M,Argnani R,Corallini A,Negrini M,Manservigi R

更新日期：2007-03-01 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：: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：:Cationic liposomes provide a means to introduce genes into cells both ex vivo and in vivo. In the past few years their use has been described in several tissues, e.g. lungs, liver, endothelium, brain. In this study we evaluated a commercially available poly-cationic liposome formulation in delivering a reporter gene i...

journal_title：Gene therapy

authors： Vitiello L,Chonn A,Wasserman JD,Duff C,Worton RG

更新日期：1996-05-01 00:00:00

abstract：:Gene transfer vectors encoding two or more genes are potentially powerful research tools and are poised to play an increasingly important role in gene therapy applications. Common strategies employed to express more than one transgene per vector include the use of multiple promoters, internal ribosome entry site (IRES...

journal_title：Gene therapy

authors： Curtin JA,Dane AP,Swanson A,Alexander IE,Ginn SL

更新日期：2008-03-01 00:00:00

abstract：:The successful development of adenovirus vectors for vaccines and gene therapy will require a better understanding of the host immune response. Using the ELISPOT assay to measure IFN-gamma-secreting CD8(+) cells, we identify immunodominant epitopes of the adenovirus hexon and DNA-binding protein in BALB/c and C57BL/6 ...

journal_title：Gene therapy

authors： McKelvey T,Tang A,Bett AJ,Casimiro DR,Chastain M

更新日期：2004-05-01 00:00:00

abstract：:Efficient gene delivery is a critical obstacle for gene therapy that must be overcome. Until current limits of gene delivery technology are solved, identification of systems with bystander effects is highly desirable. As an anticancer agent, radioactive iodine (131)I has minimal toxicity. The physical characteristics ...

journal_title：Gene therapy

authors： Mitrofanova E,Unfer R,Vahanian N,Link C

更新日期：2006-07-01 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：: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：:One factor limiting the ability to modify human repopulating hematopoietic cells genetically with retroviral vectors is the relatively low expression of the cognate viral receptor. We have tested sequential transduction of human hematopoietic cells with an adenoviral vector encoding the ecotropic retroviral receptor f...

journal_title：Gene therapy

authors： Nathwani AC,Persons DA,Stevenson SC,Frare P,McClelland A,Nienhuis AW,Vanin EF

更新日期：1999-08-01 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：:Mutations in GJB2, which codes for the gap junction (GJ) protein connexin26 (Cx26), are the most common causes of human nonsyndromic hereditary deafness. We inoculated modified adeno-associated viral (AAV) vectors into the scala media of early postnatal conditional Gjb2 knockout mice to drive exogenous Cx26 expression...

journal_title：Gene therapy

authors： Yu Q,Wang Y,Chang Q,Wang J,Gong S,Li H,Lin X

更新日期：2014-01-01 00:00:00

abstract：:Transplantation faces several major obstacles that could be overcome by expression of immunomodulatory proteins through application of gene therapy techniques. Gene therapy strategies to prolong graft survival involve gene transfer of immunosuppressive or graft-protecting molecules. Very promising results have been ob...

journal_title：Gene therapy

authors： Guillot C,Le Mauff B,Cuturi MC,Anegon I

更新日期：2000-01-01 00:00:00

abstract：:A major concern of using viral gene therapy is the potential for uncontrolled vector propagation and infection that might result in serious deleterious effects. To enhance the safety, several viral vectors, including vectors based on Sindbis virus, were engineered to lose their capability to replicate and spread after...

journal_title：Gene therapy

authors： Tseng JC,Daniels G,Meruelo D

更新日期：2009-02-01 00:00:00

abstract：:Hydrodynamic gene delivery is an attractive option for non-viral liver gene therapy, but requires evaluation of efficacy, safety and clinically applicable techniques in large animal models. We have evaluated retrograde delivery of DNA to the whole liver via the isolated segment of inferior vena cava (IVC) draining the...

journal_title：Gene therapy

authors： Fabre JW,Grehan A,Whitehorne M,Sawyer GJ,Dong X,Salehi S,Eckley L,Zhang X,Seddon M,Shah AM,Davenport M,Rela M

更新日期：2008-03-01 00:00:00

abstract：:For patients with metastatic prostate cancer, the 5-year survival rate of 31% points to a need for novel therapies and improvement of existing modalities. We propose that p53 gene therapy and chemotherapy, when combined, will provide superior tumor cell killing for the treatment of prostate carcinoma. To this end, we ...

journal_title：Gene therapy

authors： Tamura RE,Lana MG,Costanzi-Strauss E,Strauss BE

更新日期：2020-02-01 00:00:00

abstract：:Different lipids and cationic polymers were tested in vitro for their ability to transfect rabbit aortic smooth muscle cells and human endothelial cells with lacZ marker gene. Toxicity of the complexes was evaluated with MTT assay. Selected plasmid-polymer complexes with different charge ratios were then tested for in...

journal_title：Gene therapy

authors： Turunen MP,Hiltunen MO,Ruponen M,Virkamäki L,Szoka FC Jr,Urtti A,Ylä-Herttuala S

更新日期：1999-01-01 00:00:00

abstract：:Glucagon-like peptide 1 (GLP-1) and its analogue exendin-4 (Ex4) have displayed potent glucose homeostasis-modulating characteristics in type 2 diabetes (T2D). However, there are few reports of effectiveness in type 1 diabetes (T1D) therapy, where there is massive loss of beta cells. We previously described a novel GL...

journal_title：Gene therapy

authors： Soltani N,Kumar M,Glinka Y,Prud'homme GJ,Wang Q

更新日期：2007-06-01 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