Development of a nonintegrating Rev-dependent lentiviral vector carrying diphtheria toxin A chain and human TRAF6 to target HIV reservoirs.

abstract：:Recombinant human platelet-derived growth factor-BB (rhPDGF-BB) promotes soft tissue and bone healing, and is Food and Drug Administration-approved for treatment of diabetic ulcers and periodontal defects. The short half-life of topical rhPDGF-BB protein application necessitates bolus, high-dose delivery. Gene therapy...

journal_title：Gene therapy

authors： Plonka AB,Khorsand B,Yu N,Sugai JV,Salem AK,Giannobile WV,Elangovan S

更新日期：2017-01-01 00:00:00

abstract：:Adoptive cell therapy with chimeric antigen receptor (CAR)-modified T cells showed remarkable therapeutic efficacy in the treatment of leukaemia/lymphoma. However, the application to a variety of cancer entities is often constricted by the non-availability of a single chain antibody (scFv), which is usually the target...

journal_title：Gene therapy

authors： Faitschuk E,Nagy V,Hombach AA,Abken H

更新日期：2016-10-01 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：: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 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：:Although particle-mediated gene transfer using gene gun technology has been applied for gene transfer into epidermis, applications of this technology to visceral tissues have not been well investigated. Although all helium gas-driven gene gun instruments have used macrocarriers to discharge DNA-coated microprojectiles...

journal_title：Gene therapy

authors： Kuriyama S,Mitoro A,Tsujinoue H,Nakatani T,Yoshiji H,Tsujimoto T,Yamazaki M,Fukui H

更新日期：2000-07-01 00:00:00

abstract：:Integration site analysis was performed on six dogs with canine leukocyte adhesion deficiency (CLAD) that survived greater than 1 year after infusion of autologous CD34+ bone marrow cells transduced with a gammaretroviral vector expressing canine CD18. A total of 387 retroviral insertion sites (RIS) were identified in...

journal_title：Gene therapy

authors： Hai M,Adler RL,Bauer TR Jr,Tuschong LM,Gu YC,Wu X,Hickstein DD

更新日期：2008-07-01 00:00:00

abstract：:Transplantation of allogeneic cells as well as of genetically corrected autologous cells are potent approaches to restore cellular functions in patients suffering from genetic diseases. The recipient's immune responses against non-self-antigens may compromise the survival of the grafted cells. Recipients of the graft ...

journal_title：Gene therapy

authors： Bots STF,Hoeben RC

更新日期：2017-07-01 00:00:00

abstract：:To identify the intracellular barriers to efficient gene transfer, we studied the intracellular trafficking of biotinylated plasmid DNA complexed with either fluorescein-conjugated lactosylated or mannosylated polylysine by confocal microscopy. Both are known to be taken up by cystic fibrosis airway epithelial cells (...

journal_title：Gene therapy

authors： Grosse S,Tremeau-Bravard A,Aron Y,Briand P,Fajac I

更新日期：2002-08-01 00:00:00

abstract：:A recombinant adeno-associated virus (rAAV) vector was used to overexpress the anti-apoptotic Bcl-2-family protein, BCL-w, in rat brain. Three weeks after injecting the vector into cerebral cortex and striatum on one side, temporary focal ischemia was induced by occlusion of the ipsilateral middle cerebral artery for ...

journal_title：Gene therapy

authors： Sun Y,Jin K,Clark KR,Peel A,Mao XO,Chang Q,Simon RP,Greenberg DA

更新日期：2003-01-01 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：: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：: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：:The mouse is widely used as a model for DNA therapy and vaccination even though the efficiency of DNA delivery in higher mammals and humans is much less. The human APOBEC3 (A3) enzymes impact viral genomes by cytidine deamination, which introduces multiple uridine residues into single-stranded DNA, a process known as ...

journal_title：Gene therapy

authors： Kostrzak A,Henry M,Demoyen PL,Wain-Hobson S,Vartanian JP

更新日期：2015-01-01 00:00:00

abstract：:Cationic lipid-based delivery systems such as lipoplexes or stabilized plasmid-lipid particles (SPLP) represent a safer alternative to viral systems for gene therapy applications. We studied the impact of cell cycle status on the efficiency of transfection of human ovarian carcinoma tumor cells using two cationic-lipi...

journal_title：Gene therapy

authors： Mortimer I,Tam P,MacLachlan I,Graham RW,Saravolac EG,Joshi PB

更新日期：1999-03-01 00:00:00

abstract：:Chronic infection with hepatitis B virus (HBV) puts individuals at high risk for complicating cirrhosis and liver cancer, but available treatment to counter the virus rarely eliminates infection. Although harnessing RNA interference (RNAi) to silence HBV genes has shown the potential, achieving efficient and durable s...

journal_title：Gene therapy

authors： Ivacik D,Ely A,Ferry N,Arbuthnot P

更新日期：2015-02-01 00:00:00

abstract：:Hypoxia initiates an adaptive physiological response in all organisms and plays a role in the pathogenesis of several human diseases. The hypoxia/HIF-inducible factor-1 (HIF-1) transcription factor mediates transcriptional responses to hypoxia by binding to a cis-acting hypoxia-responsive element (HRE) present within ...

journal_title：Gene therapy

authors： Post DE,Van Meir EG

更新日期：2001-12-01 00:00:00

abstract：:Galactose(alpha1,3)galactose on the surface of cells of non-primate organs is the major xenoantigen responsible for hyperacute rejection in xenotransplantation. The antigen is synthesised by (alpha1, 3)galactosyl transferase. Humans lack this enzyme and their serum contains high levels of pre-existing natural antibody...

journal_title：Gene therapy

authors： Jäger U,Takeuchi Y,Porter C

更新日期：1999-06-01 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：:Solid tumors meet their demands for nascent blood vessels and increased glycolysis, to combat hypoxia, by activating multiple genes involved in angiogenesis and glucose metabolism. Hypoxia inducible factor-1 (HIF-1) is a constitutively expressed basic helix-loop-helix transcription factor, formed by the assembly of HI...

journal_title：Gene therapy

authors： Sun X,Kanwar JR,Leung E,Lehnert K,Wang D,Krissansen GW

更新日期：2001-04-01 00:00:00

abstract：:The ability to express recombinant genes in the coronary vasculature and the myocardium holds promise for the treatment of a number of acquired and inherited cardiovascular diseases. Previous in vivo gene transfer approaches in the heart have been limited by relatively low efficiencies of gene transduction. In this re...

journal_title：Gene therapy

authors： Barr E,Carroll J,Kalynych AM,Tripathy SK,Kozarsky K,Wilson JM,Leiden JM

更新日期：1994-01-01 00:00:00

abstract：:Virotherapy is currently being developed for many different types of viruses including replication-competent murine leukaemia virus (MLV) as a novel tool in cancer therapy. However, there is the risk of insertional mutagenesis associated with this virus, making careful preclinical studies necessary before its first ap...

journal_title：Gene therapy

authors： Duerner LJ,Schwantes A,Schneider IC,Cichutek K,Buchholz CJ

更新日期：2008-11-01 00:00:00

abstract：:Cell surface targeting of recombinant adeno-associated virus (rAAV) vectors is an attractive strategy to modify AAV's natural tropism. As modification of the capsid surface is likely to affect the mechanism of vector internalization and consequently the vector's intracellular fate, we investigated early steps in cell ...

journal_title：Gene therapy

authors： Uhrig S,Coutelle O,Wiehe T,Perabo L,Hallek M,Büning H

更新日期：2012-02-01 00:00:00

abstract：:X-linked chronic granulomatous disease (X-CGD) is an inherited immunodeficiency with absent phagocyte NADPH-oxidase activity caused by defects in the gene-encoding gp91(phox). Here, we evaluated strategies for less intensive conditioning for gene therapy of genetic blood disorders without selective advantage for gene ...

journal_title：Gene therapy

authors： Sadat MA,Dirscherl S,Sastry L,Dantzer J,Pech N,Griffin S,Hawkins T,Zhao Y,Barese CN,Cross S,Orazi A,An C,Goebel WS,Yoder MC,Li X,Grez M,Cornetta K,Mooney SD,Dinauer MC

更新日期：2009-12-01 00:00:00

abstract：:Injury-induced neointimal development results from migration and proliferation of vascular smooth muscle cells (SMC). Cell migration requires controlled proteolytic degradation of extracellular matrix surrounding the cell. Plasmin is a major contributor to this process by degrading various matrix proteins directly, or...

journal_title：Gene therapy

authors： Lamfers ML,Lardenoye JH,de Vries MR,Aalders MC,Engelse MA,Grimbergen JM,van Hinsbergh VW,Quax PH

更新日期：2001-04-01 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：:In this study, we describe a simple system in which human keratinocytes can be redirected to an alternative differentiation pathway. We transiently transfected freshly isolated human skin keratinocytes with the single transcription factor OCT4. Within 2 days these cells displayed expression of endogenous embryonic gen...

journal_title：Gene therapy

authors： Racila D,Winter M,Said M,Tomanek-Chalkley A,Wiechert S,Eckert RL,Bickenbach JR

更新日期：2011-03-01 00:00:00

abstract：:There is currently an urgent need to develop efficient gene-delivery systems for the lung that are free of inflammatory effects. The LID vector is a synthetic gene delivery system, comprised of lipofectin (L), an integrin-targeting peptide (I) and DNA (D) that has previously been shown to have high transfection effici...

journal_title：Gene therapy

authors： Jenkins RG,Meng QH,Hodges RJ,Lee LK,Bottoms SE,Laurent GJ,Willis D,Ayazi Shamlou P,McAnulty RJ,Hart SL

更新日期：2003-06-01 00:00:00

abstract：:Alphavirus vectors based on Sindbis virus and Semliki Forest virus (SFV) were characterized as potential gene transfer vectors. Initial studies were performed using vectors engineered to transfer either lacZ or green fluorescent protein (GFP). High levels of gene transfer were achieved in human primary fibroblasts, BH...

journal_title：Gene therapy

authors： Wahlfors JJ,Zullo SA,Loimas S,Nelson DM,Morgan RA

更新日期：2000-03-01 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