In vivo targeted delivery of CD40 shRNA to mouse intestinal dendritic cells by oral administration of recombinant Sacchromyces cerevisiae.

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：: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：:We have developed a novel immunostimulatory molecule against tumor cells, composed of an anti-FcgammaRIII (CD16) scFv fused to the platelet-derived growth factor receptor (PDGFR) transmembrane region. This fusion molecule was stably expressed on the tumor cell surface and retained the ability of the parental antibody ...

journal_title：Gene therapy

authors： Gruel N,Fridman WH,Teillaud JL

更新日期：2001-11-01 00:00:00

abstract：:This study characterized gene transfer into both normal and injured adult rat dorsal spinal cord using first (E1-/E3-) or second (E1-/E2A125/E3-, temperature-sensitive; ts) generation of replication-defective adenoviral (Ad) vectors. A novel immunosuppressive regimen aimed at blocking CD4/CD45 lymphocytic receptors wa...

journal_title：Gene therapy

authors： Romero MI,Smith GM

更新日期：1998-12-01 00:00:00

abstract：:Schwann cells (SCs) in an injured peripheral nerve form pathways for regenerating axons. Although these cells initially support regeneration, SCs lose their pro-regenerative properties following a prolonged period of denervation. Gene transfer to SC can enhance their therapeutic potential. In this article, we compared...

journal_title：Gene therapy

authors： Hoyng SA,De Winter F,Gnavi S,van Egmond L,Attwell CL,Tannemaat MR,Verhaagen J,Malessy MJ

更新日期：2015-10-01 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：: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：:Ideal methods for human gene therapy will eventually include direct gene transfer to defective tissues in a patient in vivo. Toward that goal, we have used high titer, pseudotyped retroviral vectors expressing genes for the Escherichia coli beta-galactosidase (lacZ) or hepatitis B virus surface antigen (HBsAg) to infe...

journal_title：Gene therapy

authors： Miyanohara A,Yee JK,Bouic K,LaPorte P,Friedmann T

更新日期：1995-03-01 00:00:00

abstract：:Insulin gene therapy in clinical medicine is currently hampered by the inability to regulate insulin secretion in a physiological manner, the inefficiency with which the gene is delivered, and the short duration of gene expression. To address these issues, we injected the liver of streptozotocin-induced diabetic rats ...

journal_title：Gene therapy

authors： Kim YD,Park KG,Morishita R,Kaneda Y,Kim SY,Song DK,Kim HS,Nam CW,Lee HC,Lee KU,Park JY,Kim BW,Kim JG,Lee IK

更新日期：2006-02-01 00:00:00

abstract：:A number of genetic disorders are manifested in cutaneous epithelium and gene therapy approaches for treatment of such diseases are being considered. A successful gene therapy protocol requires durable and correctly targeted gene expression within the tissue. The continuous renewal and high levels of compartmentalizat...

journal_title：Gene therapy

authors： Ghazizadeh S,Doumeng C,Taichman LB

更新日期：2002-10-01 00:00:00

abstract：:Interferon-alpha (IFN-alpha) or CD80 transduction of tumor cells individually reduces tumorigenicity and enhances antitumor responses. Here, we report that the combination of IFN-alpha and CD80 cancer gene therapy in poorly immunogenic murine tumor models, the colorectal adenocarcinoma cell line MC38, and the methylch...

journal_title：Gene therapy

authors： Hiroishi K,Tüting T,Tahara H,Lotze MT

更新日期：1999-12-01 00:00:00

abstract：:Cellular immunity against cancer can be achieved with viral vector- and DNA-based immunizations. In preclinical studies, cancer vaccines are very potent, but in clinical trials these potencies are not achieved yet. Thus, a rational approach to improve cancer vaccines is warranted. We previously demonstrated that the r...

journal_title：Gene therapy

authors： Ip PP,Boerma A,Walczak M,Oosterhuis K,Haanen JB,Schumacher TN,Nijman HW,Daemen T

更新日期：2015-07-01 00:00:00

abstract：:Gene therapy of cystic fibrosis (CF) lung disease needs highly efficient delivery and long-lasting complementation of the CFTR (cystic fibrosis transmembrane conductance regulator) gene into the respiratory epithelium. The development of lentiviral vectors has been a recent advance in the field of gene transfer and th...

journal_title：Gene therapy

authors： Copreni E,Penzo M,Carrabino S,Conese M

更新日期：2004-10-01 00:00:00

abstract：:Improving the therapeutic potential of adenoviral (Ad) suicide gene therapy has become an area of intense investigation since the inception of gene therapy strategies for cancer treatment. Poor efficiency of gene transfer to target tissues has become one of the most important limitations to Ad-based gene therapy. Sinc...

journal_title：Gene therapy

authors： Lanuti M,Kouri CE,Force S,Chang M,Amin K,Xu K,Blair I,Kaiser L,Albelda S

更新日期：1999-09-01 00:00:00

abstract：:In view of our recent findings that a truncated form of the envelope (Env) glycoprotein of human immunodeficiency virus type 1 (HIV-1) was efficiently incorporated into MoMLV particles, we studied the generation of Moloney murine leukemia virus (MoMLV)/simian immunodeficiency virus (SIV) pseudotypes. Unlike HIV-1, bot...

journal_title：Gene therapy

authors： Indraccolo S,Minuzzo S,Feroli F,Mammano F,Calderazzo F,Chieco-Bianchi L,Amadori A

更新日期：1998-02-01 00:00:00

abstract：:Recombinant adeno-associated virus (rAAV) vectors are attractive candidates for the treatment of inherited and acquired retinal disease. Although rAAV vectors are well characterized in rodent models, a prerequisite to their clinical application in human patients is the thorough evaluation of their efficacy and safety ...

journal_title：Gene therapy

authors： Bainbridge JW,Mistry A,Schlichtenbrede FC,Smith A,Broderick C,De Alwis M,Georgiadis A,Taylor PM,Squires M,Sethi C,Charteris D,Thrasher AJ,Sargan D,Ali RR

更新日期：2003-08-01 00:00:00

abstract：:The number of clinical trials using gene transfer technology, either active or under discussion, is increasing rapidly. However, little information is available describing the regulatory procedures or safety specifications that must be considered before initiation of such trials in Europe. We describe the procedure us...

journal_title：Gene therapy

authors： Caplen NJ,Gao X,Hayes P,Elaswarapu R,Fisher G,Kinrade E,Chakera A,Schorr J,Hughes B,Dorin JR

更新日期：1994-03-01 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：:Replicating viruses for cancer gene therapy have beneficial antitumor effects, however, in the setting of an enzyme/prodrug system, the interactions between these viruses and the activated agents are complex. A replicating vaccinia virus expressing the cytosine deaminase gene (VVCD), which converts the prodrug 5-FC in...

journal_title：Gene therapy

authors： McCart JA,Puhlmann M,Lee J,Hu Y,Libutti SK,Alexander HR,Bartlett DL

更新日期：2000-07-01 00:00:00

abstract：:Current tools for the inhibition of microRNA (miR) function are limited to modified antisense oligonucleotides, sponges and decoy RNA molecules and none have been used to understand miR function during development. CRISPR/Cas-mediated deletion of miR sequences within the genome requires multiple chromosomal deletions ...

journal_title：Gene therapy

authors： Cao H,Yu W,Li X,Wang J,Gao S,Holton NE,Eliason S,Sharp T,Amendt BA

更新日期：2016-06-01 00:00:00

abstract：:The clinical efficacy and safety as well as the application range of gene therapy will be broadened by developing systems capable of finely modulating the expression of therapeutic genes. Transgene regulation will be crucial for maintaining appropriate levels of a gene product within the therapeutic range, thus preven...

journal_title：Gene therapy

authors： Toniatti C,Bujard H,Cortese R,Ciliberto G

更新日期：2004-04-01 00:00:00

abstract：:Citrullinemia is an autosomal recessive disorder caused by the deficiency of argininosuccinate synthetase (AS). It is characterized by elevated levels of blood citrulline and ammonia, which often results in hyperammonemic coma and early neonatal death in affected children. We have explored the use of adenoviral vector...

journal_title：Gene therapy

authors： Ye X,Whiteman B,Jerebtsova M,Batshaw ML

更新日期：2000-10-01 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：:Exogenous osteoprotegerin (OPG) gene modification appears a therapeutic strategy for osteolytic aseptic loosening. The feasibility and efficacy of a cell-based OPG gene delivery approach were investigated using a murine model of knee prosthesis failure. A titanium pin was implanted into mouse proximal tibia to mimic a...

journal_title：Gene therapy

authors： Zhang L,Jia TH,Chong AC,Bai L,Yu H,Gong W,Wooley PH,Yang SY

更新日期：2010-10-01 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：: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：:Both atherosclerosis and arterial interventions induce oxidative stress mediated in part by nicotinamide adenine dinucleotide phosphate (NADPH) oxidases that have a pivotal role in the development of neointimal hyperplasia and restenosis. For small interfering RNA (siRNA) targeting of the NOX2 (Cybb) component of the ...

journal_title：Gene therapy

authors： Li JM,Newburger PE,Gounis MJ,Dargon P,Zhang X,Messina LM

更新日期：2010-10-01 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：: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 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