Bidirectional promoter interference between two widely used internal heterologous promoters in a late-generation lentiviral construct.

abstract：:Gene modification of malignant cells to express immune stimulators (cytokines and immune costimulators) has provided the basis for a novel form of immunotherapy. Using a MPSV-based retroviral vector with hygromycin resistance gene as a selectable marker, we have studied retrovirus-mediated gene transfer of an immune c...

journal_title：Gene therapy

authors： Hirst WJ,Buggins A,Darling D,Gäken J,Farzaneh F,Mufti GJ

更新日期：1997-07-01 00:00:00

abstract：:We created Na(+)/HCO3(-) cotransporter 1 (NBCe1) p.W516* knock-in mice as a model of isolated proximal renal tubular acidosis showing early lethality associated with severe metabolic acidosis to investigate the therapeutic effects of prenatal alkalization or posttranscriptional control 124 (PTC124). NBCe1(W516*/W516*)...

journal_title：Gene therapy

authors： Fang YW,Yang SS,Chau T,Nakamura M,Yamazaki O,Seki G,Yamada H,Hsu HM,Cheng CJ,Lin SH

更新日期：2015-05-01 00:00:00

abstract：:Human immunodeficiency virus (HIV) infection ultimately leads to the destruction of the CD4+ lymphocyte subset and the onset of AIDS. In recent years, several gene therapy procedures making use of retroviral vectors that selectively target HIV susceptible cells have been proposed in order to interfere with HIV product...

journal_title：Gene therapy

authors： Lodge R,Subbramanian RA,Forget J,Lemay G,Cohen EA

更新日期：1998-05-01 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：:This report examines the commercialization of gene therapy in the context of innovation theories that posit a relationship between the maturation of a technology through its life cycle and prospects for successful product development. We show that the field of gene therapy has matured steadily since the 1980s, with th...

journal_title：Gene therapy

authors： Ledley FD,McNamee LM,Uzdil V,Morgan IW

更新日期：2014-02-01 00:00:00

abstract：:Angiogenesis plays a pivotal role in tumor growth, tissue invasion and metastasis. Endostatin is an angiogenesis inhibitor and has been shown to reduce tumor growth in animal models. However, therapy with recombinant endostatin protein was hampered by its short half-life and very-low yield of bioactive protein. We per...

journal_title：Gene therapy

authors： Li HL,Li S,Shao JY,Lin XB,Cao Y,Jiang WQ,Liu RY,Zhao P,Zhu XF,Zeng MS,Guan ZZ,Huang W

更新日期：2008-02-01 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

abstract：:We introduce a lung inflation-fixation protocol to examine the distribution and gene transfer efficiency of fluorescently tagged lipoplexes using fluorescence confocal microscopy within thick lung tissue sections. Using this technique, we tested the hypothesis that factors related to lipoplex distribution were the pre...

journal_title：Gene therapy

authors： Uyechi LS,Gagné L,Thurston G,Szoka FC Jr

更新日期：2001-06-01 00:00:00

abstract：:A stable packaging cell line (Vero/BC-F) constitutively expressing fusion (F) protein of the human parainfluenza virus type 2 (hPIV2) was established for production of the F-defective and single round-infectious hPIV2 vector in a strategy for recombinant vaccine development. The F gene expression has not evoked cytost...

journal_title：Gene therapy

authors： Ohtsuka J,Fukumura M,Tsurudome M,Hara K,Nishio M,Kawano M,Nosaka T

更新日期：2014-08-01 00:00:00

abstract：:Intraperitoneal (i.p.) recurrence of cisplatin-refractory and p53 mutant ovarian cancer is a major clinical problem, despite surgery and chemotherapy. dl1520 (ONYX-015) is an E1B-55 kDa gene-deleted adenovirus engineered selectively to replicate in and destroy cancer cells lacking functional p53. However, a correlatio...

journal_title：Gene therapy

authors： Heise C,Ganly I,Kim YT,Sampson-Johannes A,Brown R,Kirn D

更新日期：2000-11-01 00:00:00

abstract：:Coagulation factor X (FX)-binding ablated adenovirus type 5 (Ad5) vectors have been genetically engineered to ablate the interaction with FX, resulting in substantially reduced hepatocyte transduction following intravenous administration in rodents. Here, we quantify viral genomes and gene transfer mediated by Ad5 and...

journal_title：Gene therapy

authors： Alba R,Bradshaw AC,Mestre-Francés N,Verdier JM,Henaff D,Baker AH

更新日期：2012-01-01 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：:For the development of human immunodeficiency virus type 1 (HIV-1) vaccines, traditional approaches inducing virus-neutralizing antibodies have so far failed. Thus the effort is now focused on elicitation of cellular immunity. We are currently testing in clinical trials in the United Kingdom and East Africa a T-cell v...

journal_title：Gene therapy

authors： Nkolola JP,Wee EG,Im EJ,Jewell CP,Chen N,Xu XN,McMichael AJ,Hanke T

更新日期：2004-07-01 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：:Bone morphogenetic protein (BMP) adenoviral vectors for the induction of osteogenesis are being developed for the treatment of bone pathology. However, it is still unknown which BMP adenoviral vector has the highest potential to stimulate bone formation in vivo. In this study, the osteogenic activities of recombinant ...

journal_title：Gene therapy

authors： Li JZ,Li H,Sasaki T,Holman D,Beres B,Dumont RJ,Pittman DD,Hankins GR,Helm GA

更新日期：2003-09-01 00:00:00

abstract：:The bystander effect (BSE) is an interesting and important property of the herpes thymidine kinase/ganciclovir (hTK/GCV) system of gene therapy for cancer. With the BSE, not only are the hTK expressing cells killed upon ganciclovir (GCV) exposure but also neighboring wild-type tumor cells. On testing a large number of...

journal_title：Gene therapy

authors： Touraine RL,Ishii-Morita H,Ramsey WJ,Blaese RM

更新日期：1998-12-01 00:00:00

abstract：:We have designed new vectors for the construction of recombinant adenoviruses containing expression cassettes in the E1 and/or E3 regions. Using a versatile set of restriction enzymes, the cassettes are cloned into small bacterial vectors and subsequently introduced into large plasmids containing the adenoviral sequen...

journal_title：Gene therapy

authors： Danthinne X,Werth E

更新日期：2000-01-01 00:00:00

abstract：:We report on long-term delivery of an interferon-gamma (IFN gamma) inhibitory protein by intramuscular (i.m.) gene therapy. IFN gamma is a cytokine that plays an important role in many inflammatory disorders, including autoimmune insulin-dependent diabetes mellitus (IDDM) in NOD mice and (in various strains) multiple ...

journal_title：Gene therapy

authors： Prud'homme GJ,Chang Y

更新日期：1999-05-01 00:00:00

abstract：:Parkinson's disease is a prevalent progressive degenerative disorder of the elderly. There is a current need for novel therapeutic strategies because the standard levodopa pharmacotherapy is only temporarily efficacious. Recently, there have been some high-profile successful preclinical results obtained in animal mode...

journal_title：Gene therapy

authors： Manfredsson FP,Lewin AS,Mandel RJ

更新日期：2006-03-01 00:00:00

abstract：:HaCaT cells, a spontaneously immortalised, nontumorigenic keratinocyte line, were used as a more amenable model than primary keratinocytes for ex vivo-mediated gene transfer. These cells were transduced with retroviral vectors containing the factor IX cDNA under the control of a cytomegaloviral (CMV) promoter/enhancer...

journal_title：Gene therapy

authors： Page SM,Brownlee GG

更新日期：1998-03-01 00:00:00

abstract：:The efficiency with which adenoviral vectors infect airway epithelial cells in vivo is unclear despite extensive preclinical and clinical studies. Our hypothesis is that gene transfer is limited by vector internalization which is mediated by binding of a fiber with a cellular receptor and the RGD motif of the penton b...

journal_title：Gene therapy

authors： Goldman M,Su Q,Wilson JM

更新日期：1996-09-01 00:00:00

abstract：:In order to characterize protein targeting signals in polarized postmitotic cortical neurones in vitro, we have developed recombinant and amplicon type vectors derived from herpes simplex virus 1 (HSV1) to transfer genes into these cells. We examined the targeting of both bacterial proteins, which lack specific target...

journal_title：Gene therapy

authors： Lowenstein PR,Bain D,Morrison EE,Preston CM,Clissold P,Fournel S,Epstein A,Castro MG

更新日期：1994-01-01 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：:Highly active antiretroviral therapy (HAART) treatment for HIV has changed the course of AIDS in societies in which the drugs are readily available. Despite the great success of HAART, drug resistance and toxicity issues still remain a concern for some individuals. Thus, a number of investigators have been exploring o...

journal_title：Gene therapy

authors： Zhou J,Rossi JJ

更新日期：2011-12-01 00:00:00

abstract：:Antibodies have long been used in biomedical science as in vitro tools for the identification, purification and functional manipulation of target antigens; they have been exploited in vivo for diagnostic and therapeutic applications as well. Recent advances in antibody engineering have now allowed the genes encoding a...

journal_title：Gene therapy

authors： Marasco WA

更新日期：1997-01-01 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：:We recently showed that the human telomerase reverse transcriptase (hTERT) promoter induces tumor-specific Bax gene expression and selectively kills various human cancer cells both in vitro and in xenograft tumors. However, it remains unclear whether the hTERT promoter can be used to induce transgene expression in syn...

journal_title：Gene therapy

authors： Gu J,Andreeff M,Roth JA,Fang B

更新日期：2002-01-01 00:00:00

abstract：:The oncotropic phenotypes of several viruses correlate with tumor-associated deficiencies within interferon (IFN) signaling pathways. This observation formed the conceptual basis for developing oncolytic viruses deleted for viral proteins that inhibit the host IFN-dependent antiviral response, such as herpes simplex v...

journal_title：Gene therapy

authors： Sobol PT,Hummel JL,Rodrigues RM,Mossman KL

更新日期：2009-09-01 00:00:00

abstract：:Multipotent adult progenitor cells (MAPCs) are bone marrow-derived stem cells with a high growth rate suitable for therapeutical applications as three-dimensional (3D) aggregates. Combined applications of osteogenically differentiated MAPC (OD-MAPC) aggregates and adeno-associated viral vectors (AAV) in bone bioengine...

journal_title：Gene therapy

authors： Ferreira JR,Hirsch ML,Zhang L,Park Y,Samulski RJ,Hu WS,Ko CC

更新日期：2013-02-01 00:00:00

abstract：:The umbilical cord provides a rich source of primitive mesenchymal stem cells (human umbilical cord mesenchymal stem cells (HUMSCs)), which have the potential for transplantation-based treatments of Parkinson's Disease (PD). Our pervious study indicated that adenovirus-associated virus-mediated intrastriatal delivery ...

journal_title：Gene therapy

authors： Xiong N,Zhang Z,Huang J,Chen C,Zhang Z,Jia M,Xiong J,Liu X,Wang F,Cao X,Liang Z,Sun S,Lin Z,Wang T

更新日期：2011-04-01 00:00:00