Coexpression of cytidine deaminase and mutant dihydrofolate reductase by a bicistronic retroviral vector confers resistance to cytosine arabinoside and methotrexate.

abstract：:The combination of immunization strategies with gene therapy methods constitutes a powerful tool for the purpose of genetic immunization. The cutaneous microenvironment, rich in professional antigen-presenting cells and accessory cells capable of initiating and controlling the intensity of specific immune responses, m...

journal_title：Human gene therapy

authors： Larregina AT,Falo LD Jr

更新日期：2000-11-01 00:00:00

abstract：:Cotransfer of a therapeutic gene together with the human MDR1 gene provides an opportunity to increase the number of transduced marrow cells, expressing the therapeutic gene, by in vivo selection for MDR1. We have used an Lg-MDR1-IRES-neo (LgMIN) retroviral vector, containing MDR1 and neo genes, separated by the EMCV ...

journal_title：Human gene therapy

authors： Zaboikin MM,Schuening FG

更新日期：1998-10-10 00:00:00

abstract：:Unlike oncoretroviruses, lentiviral vectors can insert large genes and can target both dividing and nondividing cells; thus they hold unique promise as gene transfer agents. To enhance target range, the native lentiviral envelope glycoprotein is replaced (pseudotyped) with vesicular stomatitis virus G (VSVG), and the ...

journal_title：Human gene therapy

authors： Kumar M,Bradow BP,Zimmerberg J

更新日期：2003-01-01 00:00:00

abstract：:In the next decades, gene editing technologies are expected to be used in the treatment and prevention of human diseases. Yet, the future uses of gene editing in medicine are still unknown, including its applicability and effectiveness to the treatment and prevention of infectious diseases, cancer, and monogenic and p...

journal_title：Human gene therapy

authors： Rocha LFM,Braga LAM,Mota FB

更新日期：2020-08-01 00:00:00

abstract：:We reported that SNV-derived retroviral vectors, which display single-chain antibodies on the viral surface, enable cell type-specific gene delivery into various human cells. In particular, the SNV cell type-specific gene delivery vector system appears to be well suited to transduce genes into cells of the human hemat...

journal_title：Human gene therapy

authors： Jiang A,Fisher H,Pomerantz RJ,Dornburg R

更新日期：1999-11-01 00:00:00

abstract：:Fibroblast-like synoviocytes (FLSs) participate in the pathogenesis of rheumatoid arthritis (RA). Emerging evidence has highlighted the role of long non-coding RNA metastasis associated lung adenocarcinoma transcript 1 (MALAT1) and its potential involvement in RA. In this study, we test the hypothesis that the MALAT1 ...

journal_title：Human gene therapy

authors： Li GQ,Fang YX,Liu Y,Meng FR,Wu X,Zhang CW,Zhang Y,Liu D,Gao B

更新日期：2019-08-01 00:00:00

abstract：:The objective of this phase II investigation is to assess the safety and efficacy of a plasmid mediated approach to induce angiogenesis/arteriogenesis with the angiomatrix protein Del-1 (developmentally regulated endothelial locus 1), in subjects with intermittent claudication (IC) secondary to peripheral arterial dis...

journal_title：Human gene therapy

authors： Rajagopalan S,Olin JW,Young S,Erikson M,Grossman PM,Mendelsohn FO,Regensteiner JG,Hiatt WR,Annex BH

更新日期：2004-06-01 00:00:00

abstract：:Engineered measles virus (MV) strains deriving from the vaccine lineage represent a promising oncolytic platform and are currently being tested in phase I trials. In this study, we have demonstrated that MV strains genetically engineered to express the human sodium iodide symporter (NIS) have significant antitumor act...

journal_title：Human gene therapy

authors： Opyrchal M,Allen C,Iankov I,Aderca I,Schroeder M,Sarkaria J,Galanis E

更新日期：2012-04-01 00:00:00

abstract：:The immune response against human immunodeficiency virus type-1 (HIV-1) is believed to play a role in controlling the early stages of disease progression. The cellular immune response, in particular cytotoxic T lymphocyte (CTL) activity, may be important for eliminating virally infected cells in HIV-1-infected individ...

journal_title：Human gene therapy

authors： Laube LS,Burrascano M,Dejesus CE,Howard BD,Johnson MA,Lee WT,Lynn AE,Peters G,Ronlov GS,Townsend KS

更新日期：1994-07-01 00:00:00

abstract：:The clinical success of suicide gene therapy using herpes simplex virus type 1 thymidine kinase (TK) is largely dependent on the capacity of this enzyme to effectively induce the death of bystander cells. We have shown that fusion of TK to an 11-amino acid peptide from the basic domain of the human immunodeficiency vi...

journal_title：Human gene therapy

authors： Tasciotti E,Giacca M

更新日期：2005-12-01 00:00:00

abstract：:Chronic inflammation in tibialis anterior muscles of mdx mice was produced by a single injection of a recombinant adenovirus vector (AV) expressing an immunogenic beta-galactosidase (beta-gal). In regions of intense beta-gal staining, mononuclear infiltrates abounded, and muscle fibers showed strong extrasynaptic utro...

journal_title：Human gene therapy

authors： Waheed I,Gilbert R,Nalbantoglu J,Guibinga GH,Petrof BJ,Karpati G

更新日期：2005-04-01 00:00:00

abstract：:A phase I clinical trial was conducted in which recombinant adenovirus containing the cystic fibrosis trans-membrane regulator (CFTR) (Ad2/CFTR) was administered by bronchoscopic instillation or aerosolization to the lungs of cystic fibrosis (CF) patients. In this paper, we evaluate the efficiency of Ad2/CFTR-mediated...

journal_title：Human gene therapy

authors： Perricone MA,Morris JE,Pavelka K,Plog MS,O'Sullivan BP,Joseph PM,Dorkin H,Lapey A,Balfour R,Meeker DP,Smith AE,Wadsworth SC,St George JA

更新日期：2001-07-20 00:00:00

abstract：:NKG2D ligands (NKG2DLs) are widely expressed on ovarian cancers to various degrees, making them attractive targets for immunotherapy. Here, we applied a chimeric antigen receptor (CAR) approach for the targeting of NKG2DLs expressed on human ovarian cancer cells and evaluated the impact of pharmacological upregulation...

journal_title：Human gene therapy

authors： Song DG,Ye Q,Santoro S,Fang C,Best A,Powell DJ Jr

更新日期：2013-03-01 00:00:00

abstract：:Adrenomedullin (AM) has been shown to protect against ischemia/reperfusion-induced myocardial infarction and apoptosis. In the present study, we examined the potential neuroprotective action of delayed AM gene transfer in cerebral ischemia. Three days after a 1-hr occlusion of the middle cerebral artery (MCAO), rats w...

journal_title：Human gene therapy

authors： Xia CF,Yin H,Borlongan CV,Chao J,Chao L

更新日期：2004-12-01 00:00:00

abstract：:Cell encapsulation offers a safe and manufacturable method for the systemic delivery of therapeutic proteins from genetically engineered cells. However, control of dose delivery remains a major issue with regard to clinical application. We generated populations of immortalized murine NIH 3T3 fibroblasts that secrete m...

journal_title：Human gene therapy

authors： Serguera C,Bohl D,Rolland E,Prevost P,Heard JM

更新日期：1999-02-10 00:00:00

abstract：:Interleukin-12 (IL-12) is a cytokine that exhibits pleiotropic effects on lymphocytes and natural killer cells and has been shown to have promise for the immunotherapy of cancer. The combination of the immune costimulatory molecule B7.1 and IL-12 has been shown to be synergistic for T cell activation. By transfecting ...

journal_title：Human gene therapy

authors： Anderson R,Macdonald I,Corbett T,Hacking G,Lowdell MW,Prentice HG

更新日期：1997-06-10 00:00:00

abstract：:The enzyme hypoxanthine-guanine phosphoribosyltransferase (HGPRT) expressed by the parasite Trypanosoma brucei (Tb) can convert allopurinol, a purine analogue, to corresponding nucleotides with greater efficiency than its human homologue. We have developed a retroviral system that expresses the parasitic enzyme and te...

journal_title：Human gene therapy

authors： Trudeau C,Yuan S,Galipeau J,Benlimame N,Alaoui-Jamali MA,Batist G

更新日期：2001-09-01 00:00:00

abstract：:In vitro delivery of interferon-alpha (IFN-alpha) to cultured human monocytes by means of a replication-incompetent herpesvirus vector inhibits human immunodeficiency virus (HIV) replication. To explore the possibility of IFN-alpha gene delivery by vector-infected human monocytes, monocytes were isolated and the cultu...

journal_title：Human gene therapy

authors： Weir JP,Dacquel EJ,Aronovitz J

更新日期：1996-07-10 00:00:00

abstract：:Extracellular vesicles (EVs) being released from two adjacent adeno-associated virus serotype 1 (AAV1)-producing 293T cells are shown by electron microscopy. We have shown that AAV vectors can associate with EVs and enter the media. Furthermore, we have recently reported that EV-associated AAV has robust gene delivery...

journal_title：Human gene therapy

authors： Fitzpatrick Z,György B,Skog J,Maguire CA

更新日期：2014-09-01 00:00:00

abstract：:Administration of plasmid/lipid complexes to the lung airways for the treatment of metastatic pulmonary diseases represents a new strategy of gene therapy. In this study we present evidence that intratracheal administration of a plasmid encoding murine IL-12 complexed with N-[1-(2,3-dioleyloxy)propyl)-N,N,N-trimethyla...

journal_title：Human gene therapy

authors： Blezinger P,Freimark BD,Matar M,Wilson E,Singhal A,Min W,Nordstrom JL,Pericle F

更新日期：1999-03-20 00:00:00

abstract：:Ornithine transcarbamylase deficiency (OTCD) is an inborn error of urea synthesis that has been considered as a model for liver-directed gene therapy. Current treatment has failed to avert a high mortality or morbidity from hyperammonemic coma. Restoration of enzyme activity in the liver should suffice to normalize me...

journal_title：Human gene therapy

authors： Raper SE,Yudkoff M,Chirmule N,Gao GP,Nunes F,Haskal ZJ,Furth EE,Propert KJ,Robinson MB,Magosin S,Simoes H,Speicher L,Hughes J,Tazelaar J,Wivel NA,Wilson JM,Batshaw ML

更新日期：2002-01-01 00:00:00

abstract：:Recombinant adeno-associated virus (rAAV)-mediated gene transfer has shown promise for treating diseases in various animal models including the mdx mouse model of Duchenne muscular dystrophy (DMD). In many cases, however, preclinical studies in inbred mice have not successfully predicted human clinical responses. To a...

journal_title：Human gene therapy

authors： Wang Z,Allen JM,Riddell SR,Gregorevic P,Storb R,Tapscott SJ,Chamberlain JS,Kuhr CS

更新日期：2007-01-01 00:00:00

abstract：:Serum-induced inactivation of retroviruses is the most critical limitation for in vivo gene transfer therapy. To solve this problem, we searched for reagents that protect retroviruses from inactivation. The effects of the protease inhibitors FOY-007 and FOY-305 and of an inhibitor of the complement pathway FUT-175, al...

journal_title：Human gene therapy

authors： Miyao Y,Ikenaka K,Kishima H,Tamura M,Nakamura K,Kurumi M,Hayakawa T,Shimizu K

更新日期：1997-09-01 00:00:00

abstract：:Diabetes mellitus derives from either insulin deficiency (type I) or resistance (type II). Homozygous mutations in the insulin receptor (IR) gene cause the rare leprechaunism and Rabson-Mendenhall syndromes, severe forms of hyperinsulinemic insulin resistance for which no therapy is currently available. Systems have b...

journal_title：Human gene therapy

authors： Cotugno G,Pollock R,Formisano P,Linher K,Beguinot F,Auricchio A

更新日期：2004-11-01 00:00:00

abstract：:In vivo electroporation of plasmid DNA (DNA-EP) is an efficient and safe method for vaccines. It results in increased DNA uptake, enhances protein expression, and augments immune responses to the target antigen in a variety of species. To further improve the efficacy of DNA-EP, we evaluated small interfering RNA (siRN...

journal_title：Human gene therapy

authors： Dharmapuri S,Aurisicchio L,Biondo A,Welsh N,Ciliberto G,La Monica N

更新日期：2009-06-01 00:00:00

abstract：:Recent advances in genome sequencing have greatly improved our ability to understand and identify the causes of genetic diseases. However, there remains an urgent need for innovative, safe, and effective treatments for these diseases. CRISPR-based genome editing systems have become important and powerful tools in the ...

journal_title：Human gene therapy

authors： Carlaw TM,Zhang LH,Ross CJD

更新日期：2020-08-01 00:00:00

abstract：:pZIG(hGCSFR) is a retroviral vector that can co-express two genes and also provides alternative selection markers. This retroviral vector has been constructed to incorporate an internal ribosome entry site (IRES) element to co-express two exogenous genes in mammalian cells. Two marker/selection genes have been cloned ...

journal_title：Human gene therapy

authors： Saleh M

更新日期：1997-05-20 00:00:00

abstract：:The ability to control proliferation of grafted cells in the heart and consequent graft size could dramatically improve the efficacy of cell therapies for cardiac repair. To achieve targeted graft cell proliferation, we created a chimeric receptor (F36Vfgfr-1) composed of a modified FK506-binding protein (F36V) fused ...

journal_title：Human gene therapy

authors： Stevens KR,Rolle MW,Minami E,Ueno S,Nourse MB,Virag JI,Reinecke H,Murry CE

更新日期：2007-05-01 00:00:00

abstract：:Hereditary tyrosinemia type 1 (HT1) is an autosomal recessive disorder caused by deficiency of fumarylacetoacetate hydrolase (FAH). It has been previously shown that ex vivo hepatocyte-directed gene therapy using an integrating lentiviral vector to replace the defective Fah gene can cure liver disease in small- and la...

journal_title：Human gene therapy

authors： VanLith C,Guthman R,Nicolas CT,Allen K,Du Z,Joo DJ,Nyberg SL,Lillegard JB,Hickey RD

更新日期：2018-11-01 00:00:00

abstract：:Approaches to alter the native tropism of adenoviruses (Ads) are beneficial to increase their efficacy and safety profile. Liver tropism is important with regard to potential clinical toxicity in humans. Ad5/3 chimeras in which the Ad5 knob is substituted by the Ad3 knob, such as Ad5/3luc1, have been recently shown to...

journal_title：Human gene therapy

authors： Breidenbach M,Rein DT,Wang M,Nettelbeck DM,Hemminki A,Ulasov I,Rivera AR,Everts M,Alvarez RD,Douglas JT,Curiel DT

更新日期：2004-05-01 00:00:00