Tetracycline-inducible transgene expression mediated by a single AAV vector.

abstract：:To achieve high transgene expression in the liver, we have compared the reporter gene expression among various murine retroviral long terminal repeats (LTRs) or leader sequences in vitro. Transient reporter gene expression assays revealed the highest gene expression by the polycythemic strain of spleen focus-forming v...

journal_title：Gene therapy

authors： Ohnishi N,Itoh K,Itoh Y,Baum C,Higashitsuji H,Yamaguchi K,Tsuji T,Okanoue T,Fujita J

更新日期：2002-02-01 00:00:00

abstract：:In this study, self-inactivating (SIN) retroviral vectors based on feline foamy virus (FFV) were constructed and analysed. The FFV SIN vectors were devoid of the core FFV long terminal repeat promoter plus upstream sequences but contained all structural and regulatory genes. This design allowed sensitive detection of ...

journal_title：Gene therapy

authors： Bastone P,Löchelt M

更新日期：2004-03-01 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：:The potential of gene therapy for treatment of lung disease remains unrealised. Early model systems often resulted in promising efficiency of gene transfer, only to prove irreproducible in the clinic. While problems such as induction of host immune responses and duration of expression also need to be addressed, it is ...

journal_title：Gene therapy

authors： McBride S,Rannie D,Harrison DJ

更新日期：2000-04-01 00:00:00

abstract：:To evaluate the use of HSV-based vectors for arthritis gene therapy we have constructed a first-generation, ICP4 deficient, replication defective herpes simplex virus (HSV) vector (S/0-) and a second-generation HSV vector derivative (T/0-) deficient for the immediate-early genes ICP4, 22 and 27, each carrying a solubl...

journal_title：Gene therapy

authors： Oligino T,Ghivizzani S,Wolfe D,Lechman E,Krisky D,Mi Z,Evans C,Robbins P,Glorioso J

更新日期：1999-10-01 00:00:00

abstract：:Dendritic cells (DC) are among the most potent antigen-presenting cells known and play an important role in the initiation of antigen-specific T-lymphocyte responses. Several recent studies have demonstrated that DC expressing vector-encoded tumor-associated antigens can induce protective and therapeutic immunity in m...

journal_title：Gene therapy

authors： Diao J,Smythe JA,Smyth C,Rowe PB,Alexander IE

更新日期：1999-05-01 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：:Advances in islet transplantation have encouraged efforts to create alternative insulin-secreting cells that overcome limitations associated with current therapies. We have recently demonstrated durable correction of murine and porcine diabetes by syngeneic and autologous implantation, respectively, of primary hepatoc...

journal_title：Gene therapy

authors： Chen NK,Tan SY,Udolph G,Kon OL

更新日期：2010-05-01 00:00:00

abstract：:The pseudorabies virus (PRV) is a swine alpha herpes virus that is widely used as a neural tracer because of its marked neurotropism and transneuronal transmissibility (Card et al., 1991, 1992; Strack and Loewy 1990). PRV has been used to retrogradely identify spinal cord and brainstem connections to various periphera...

journal_title：Gene therapy

authors： Jasmin L,Tarczy-Hornoch K,Wang H,Levine JD,Basbaum AI

更新日期：1994-01-01 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：:We have used a particular folate receptor, which is overexpressed in tumor cells, for targeted DNA delivery into these cell types. This folate receptor internalizes folate through caveolae by a process named potocytosis, which is distinct from endocytosis, through clathrin-coated pits. When folate conjugated to poly-L...

journal_title：Gene therapy

authors： Gottschalk S,Cristiano RJ,Smith LC,Woo SL

更新日期：1994-05-01 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：:The canine is the most important large animal model for testing novel hemophilia A (HA) treatment. It is often necessary to use canine factor VIII (cFIII) gene or protein for the evaluation of HA treatment in the canine model. However, different biological properties between cFVIII and human FVIII (hFVIII) indicated t...

journal_title：Gene therapy

authors： Wang Q,Dong B,Firrman J,Wu W,Roberts S,Moore AR,Liu LS,Chin MP,Diao Y,Kost J,Xiao W

更新日期：2016-07-01 00:00:00

abstract：:We tested the influence of overexpression of arylsulfatase A (ASA) on the activity of other sulfatases in fibroblasts from patients with metachromatic leukodystrophy (MLD). We demonstrated that the overexpression of ASA reduces the activity of various sulfatases by a small amount but does not induce an accumulation of...

journal_title：Gene therapy

authors： Ohashi T,Matalon R,Barranger JA,Eto Y

更新日期：1995-08-01 00:00:00

abstract：:Inadequate antiprotease activity in the lungs due to alpha-1-antitrypsin (A1AT) deficiency is a factor of early-onset emphysema. We propose a new approach to gene therapy that involves the intratracheal delivery of macrophages expressing human A1AT (hA1AT). Recombinant adeno-associated virus (rAAV) plasmids encoding t...

journal_title：Gene therapy

authors： Zhang D,Wu M,Nelson DE,Pasula R,Martin WJ 2nd

更新日期：2003-12-01 00:00:00

abstract：:The GM2-gangliosidoses are neurological diseases causing premature death, thus developing effective treatment protocols is urgent. GM2-gangliosidoses result from deficiency of a lysosomal enzyme β-hexosaminidase (Hex) and subsequent accumulation of GM2 gangliosides. Genetic changes in HEXA, encoding the Hex α subunit,...

journal_title：Gene therapy

authors： Ou L,Przybilla MJ,Tăbăran AF,Overn P,O'Sullivan MG,Jiang X,Sidhu R,Kell PJ,Ory DS,Whitley CB

更新日期：2020-05-01 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：:Complexes of DNA and cationic lipid offer potential advantages for gene transfer to airway epithelia. However, we found that application of DNA-lipid (DMRIE-DOPE) complexes to primary cultures of human ciliated airway epithelia or explants of rabbit trachea generated only low levels of gene transfer. In contrast, when...

journal_title：Gene therapy

authors： Fasbender A,Zabner J,Zeiher BG,Welsh MJ

更新日期：1997-11-01 00:00:00

abstract：:Cell transplantation holds promise as a potential treatment for cardiac dysfunction. Our group has isolated populations of murine skeletal muscle-derived stem cells (MDSCs) that exhibit stem cell-like properties. Here, we investigated the fate of MDSCs after transplantation into the hearts of dystrophin-deficient mdx ...

journal_title：Gene therapy

authors： Payne TR,Oshima H,Sakai T,Ling Y,Gharaibeh B,Cummins J,Huard J

更新日期：2005-08-01 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：:Naturally occurring drug resistance genes of human origin can be exploited for selection of genetically engineered cells co-expressing a desired therapeutic transgene. Their non-immunogenicity in clinical applications would be a major asset. Human cytidine deaminase (hCD) is a chemoresistance gene that inactivates cyt...

journal_title：Gene therapy

authors： Eliopoulos N,Al-Khaldi A,Beauséjour CM,Momparler RL,Momparler LF,Galipeau J

更新日期：2002-04-01 00:00:00

abstract：:Advanced prostate cancer (PC) still remains incurable. Novel immunogene therapy shows promise as treatment strategy that can target both localized and metastasized PC. In this study, we have developed a PC-specific oncolytic adenovirus (Ad-PL-PPT-E1A) armed with fusion gene of prostate-specific antigen and CD40 ligand...

journal_title：Gene therapy

authors： Yang YF,Xue SY,Lu ZZ,Xiao FJ,Yin Y,Zhang QW,Wu CT,Wang H,Wang LS

更新日期：2014-08-01 00:00:00

abstract：:Confinement of transgene expression to target cells is highly desirable in gene therapy. Current strategies of transcriptional targeting to tumors usually rely on tissue-specific promoters to control gene expression. However, such promoters generally have much lower activity than the constitutive viral promoters. We h...

journal_title：Gene therapy

authors： Fu X,Meng F,Tao L,Jin A,Zhang X

更新日期：2003-08-01 00:00:00

abstract：:As clinical gene therapy has progressed toward realizing its potential, concern over misuse of the technology to enhance performance in athletes is growing. Although 'gene doping' is banned by the World Anti-Doping Agency, its detection remains a major challenge. In this study, we developed a methodology for direct de...

journal_title：Gene therapy

authors： Baoutina A,Coldham T,Bains GS,Emslie KR

更新日期：2010-08-01 00:00:00

abstract：:In-stent restenosis results exclusively from neointimal hyperplasia due to mechanical injury and a foreign body response to the prosthesis. Inflammation mediated by monocyte chemoattractant protein-1 (MCP-1) might therefore underlie in-stent restenosis. We recently devised a new strategy for anti-MCP-1 gene therapy by...

journal_title：Gene therapy

authors： Ohtani K,Usui M,Nakano K,Kohjimoto Y,Kitajima S,Hirouchi Y,Li XH,Kitamoto S,Takeshita A,Egashira K

更新日期：2004-08-01 00:00:00

abstract：:Glucagon-like peptide 1 (GLP-1) is released from neuroendocrine cells in the intestine in the postprandial state and augments glucose-stimulated insulin secretion from pancreatic beta cells. To develop non-beta cells that exhibit physiologically regulated insulin secretion, we coexpressed the GLP-1 receptor and human ...

journal_title：Gene therapy

authors： Wu L,Nicholson W,Wu CY,Xu M,McGaha A,Shiota M,Powers AC

更新日期：2003-09-01 00:00:00

abstract：:The possibility of achieving multiple systemic expression of human interferon-beta in mice upon repeated intravenous administration of cationic liposome-DNA complex (lipoplex) was investigated. Lipoplexes containing the pentammonio lipid pcTG90 were first optimized by selecting the most efficient ratio of pcTG90 to ph...

journal_title：Gene therapy

authors： Meyer O,Schughart K,Pavirani A,Kolbe HV

更新日期：2000-09-01 00:00:00

abstract：:Glioblastoma multiforme (GBM) is one of the most formidable brain tumors with a mean survival period of approximately 12 months. To date, a combination of radiotherapy and chemotherapy with an oral alkylating agent, temozolomide (TMZ), has been used as first-line therapy for glioma. However, the efficacy of chemothera...

journal_title：Gene therapy

authors： Kato T,Natsume A,Toda H,Iwamizu H,Sugita T,Hachisu R,Watanabe R,Yuki K,Motomura K,Bankiewicz K,Wakabayashi T

更新日期：2010-11-01 00:00:00

abstract：:Both replication-incompetent and replication-selective adenoviruses are being developed for the treatment of cancer and other diseases. Concerns have been raised about the safety of intra-vascular adenovirus administration following a patient death on a clinical trial with a replication-defective adenovirus. In additi...

journal_title：Gene therapy

authors： Reid T,Galanis E,Abbruzzese J,Sze D,Andrews J,Romel L,Hatfield M,Rubin J,Kirn D

更新日期：2001-11-01 00:00:00

abstract：:Development of efficient short-term gene transfer technologies for embryonic stem (ES) cells is urgently needed for various existing and new ES cell-based research strategies. In this study, we present a highly efficient, nonviral non-DNA technology for genetic loading of mouse ES cells based on electroporation of def...

journal_title：Gene therapy

authors： Ponsaerts P,Brown JP,Van den Plas D,Van den Eeden L,Van Bockstaele DR,Jorens PG,Van Tendeloo VF,Merregaert J,Singh PB,Berneman ZN

更新日期：2004-11-01 00:00:00