Simultaneous targeted alteration of the tyrosinase and c-kit genes by single-stranded oligonucleotides.

abstract：:Genetic modification of tumor cells with the gene for the B7.1 or with the genes for cytokines results in increased tumor cell immunogenicity. In the work reported here, immunization of naive animals with either B7.1 or gamma-IFN gene-modified MCA106 tumor cells effectively protects the host from subsequent challenge ...

journal_title：Gene therapy

authors： Yang S,Vervaert CE,Seigler HF,Darrow TL

更新日期：1999-02-01 00:00:00

abstract：:Polyethylenimine (PEI) has been studied as an efficient nonviral gene transfer vector. Here, we report the biodistribution fates and safety of plasmid DNA intravenously administered in PEI complexes. Using pCMVbeta as a model gene, the biodistribution of plasmid DNA was measured by quantitative polymerase chain reacti...

journal_title：Gene therapy

authors： Oh YK,Kim JP,Yoon H,Kim JM,Yang JS,Kim CK

更新日期：2001-10-01 00:00:00

abstract：:We previously demonstrated that recombinant adeno-associated virus vectors based on serotype 2 (rAAV2) can direct transgene expression in salivary gland cells in vitro and in vivo. However, it is not known how other rAAV serotypes perform when infused into salivary glands. The capsids of serotypes 4 and 5 are distinct...

journal_title：Gene therapy

authors： Katano H,Kok MR,Cotrim AP,Yamano S,Schmidt M,Afione S,Baum BJ,Chiorini JA

更新日期：2006-04-01 00:00:00

abstract：:Replication defective vectors derived from simple retroviruses or the more complex genomes of lentiviruses continue to offer the advantages of long-term expression, cell and tissue specific tropism, and large packaging capacity for the delivery of therapeutic genes. The occurrence of adverse events caused by insertion...

journal_title：Gene therapy

authors： Sinn PL,Sauter SL,McCray PB Jr

更新日期：2005-07-01 00:00:00

abstract：:Rhesus macaque tripartite motif (TRIM)5alpha potently inhibits early stages of human immunodeficiency virus (HIV)-1 replication, while the human orthologue has little effect on this virus. We used PCR-based random mutagenesis to construct a large library of human TRIM5alpha variants containing mutations in the PRYSPRY...

journal_title：Gene therapy

authors： Pham QT,Bouchard A,Grütter MG,Berthoux L

更新日期：2010-07-01 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：:Constitutive expression of short hairpin RNAs (shRNAs) may cause cellular toxicity in vivo and using microRNA (miRNA) scaffolds can circumvent this problem. Previously, we have shown that embedding small interfering RNA sequences targeting apolipoprotein B100 (ApoB) in shRNA (shApoB) or miRNA (miApoB) scaffolds result...

journal_title：Gene therapy

authors： Maczuga P,Verheij J,van der Loos C,van Logtenstein R,Hooijer G,Martier R,Borel F,Lubelski J,Koornneef A,Blits B,van Deventer S,Petry H,Konstantinova P

更新日期：2014-01-01 00:00:00

abstract：:Oncolytic viruses hold much promise as novel therapeutic agents that can be combined with conventional therapeutic modalities. Measles virus (MV) is known to enter cells using the signaling lymphocyte activation molecule (SLAM), which is expressed on cells of the immune system. Although human breast cancer cell lines ...

journal_title：Gene therapy

authors： Sugiyama T,Yoneda M,Kuraishi T,Hattori S,Inoue Y,Sato H,Kai C

更新日期：2013-03-01 00:00:00

abstract：:Lentiviral vectors pseudotyped with the baculovirus envelope protein GP64 transduce primary cultures of human airway epithelia (HAE) at their apical surface. Our goal in this study was to harness a directed evolution approach to develop a novel envelope glycoprotein with increased transduction properties for HAE. Usin...

journal_title：Gene therapy

authors： Sinn PL,Hwang BY,Li N,Ortiz JLS,Shirazi E,Parekh KR,Cooney AL,Schaffer DV,McCray PB Jr

更新日期：2017-10-01 00:00:00

abstract：:Gene transfer complexes containing poly-L-lysine (poly-K) and DNA with ligands directed at the serpin enzyme complex receptor (sec-R) deliver reporter genes to receptor-bearing cells in vivo. Expression lasts for about 30 days, when complexes containing long-chain poly-K are used. Extending the duration of expression ...

journal_title：Gene therapy

authors： Ziady AG,Kim J,Colla J,Davis PB

更新日期：2004-09-01 00:00:00

abstract：:The CMV promoter drives high transgene expression and is one of the most commonly used promoters for gene transfer. Tissue-specific mammalian promoters provide an alternative, and it would be useful to have a system to directly compare them to viral promoters free from potential confounding vector-related effects. In ...

journal_title：Gene therapy

authors： Kachi S,Esumi N,Zack DJ,Campochiaro PA

更新日期：2006-05-01 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：:Chimeric antigen receptor (CAR) T-cell therapies have demonstrated durable and potentially curative therapeutic efficacy against B-cell leukemia in clinical trials. A CAR strategy can target any tumor surface antigens as long as an antigen-binding receptor can be generated. New CARs that target solid tumors and have t...

journal_title：Gene therapy

authors： Wu MR,Zhang T,DeMars LR,Sentman CL

更新日期：2015-08-01 00:00:00

abstract：:Earlier this laboratory constructed a herpes simplex virus 1 recombinant (R5111) that carries a IL13 ligand inserted into glycoprotein D and can enter cells via the IL13Ralpha2 receptor commonly expressed on the surface of malignant glioma cells. In this report, we describe the properties of two recombinant viruses ca...

journal_title：Gene therapy

authors： Kamiyama H,Zhou G,Roizman B

更新日期：2006-04-01 00:00:00

abstract：:Excessive activated T-cell proliferation was observed in vivo in one patient after an anti-CD19-chimeric antigen receptor (CAR) T-cell infusion. The patient, who had chemotherapy refractory and CD19+ diffuse large B-cell lymphoma (DLBCL), received an anti-CD19 CAR T-cell infusion following conditioning chemotherapy (f...

journal_title：Gene therapy

authors： Zhang WY,Liu Y,Wang Y,Nie J,Guo YL,Wang CM,Dai HR,Yang QM,Wu ZQ,Han WD

更新日期：2018-06-01 00:00:00

abstract：:Retroviral vectors are effective shuttle systems by introducing therapeutically relevant genes stably into the genome of proliferating cells. The majority of vectors applied for research or clinical applications use neomycin for cell selection and identification. To circumvent the time consuming and potentially toxic ...

journal_title：Gene therapy

authors： Machl AW,Planitzer S,Kubbies M

更新日期：1997-04-01 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：: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：:Several of the current techniques for transfer of both oligonucleotide and plasmid DNA into the myocardium are impaired by low efficiency and toxicity. To improve gene transfer techniques, especially into the whole heart, a gene transfer method involving liposome in conjunction with a viral envelope (HVJ-liposome) was...

journal_title：Gene therapy

authors： Sawa Y,Kaneda Y,Bai HZ,Suzuki K,Fujimoto J,Morishita R,Matsuda H

更新日期：1998-11-01 00:00:00

abstract：:The expression of naked plasmid DNA coding for firefly luciferase (pRSVluc) or a secreted protein, human-alpha-1-antitrypsin (pRcCMVhAAT) in mouse skeletal muscle was characterized following administration by an improved intramuscular injection technique. Injection guided by intense illumination along the longitudinal...

journal_title：Gene therapy

authors： Levy MY,Barron LG,Meyer KB,Szoka FC Jr

更新日期：1996-03-01 00:00:00

abstract：:For certain gene therapy applications, the simultaneous delivery of multiple genes would allow for novel therapies. In the case of adeno-associated virus (AAV) vectors, the limited packaging capacity greatly restricts current methods of carrying multiple transgene cassettes. To address this issue, a recombinant AAV (r...

journal_title：Gene therapy

authors： Foti SB,Samulski RJ,McCown TJ

更新日期：2009-11-01 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：:Acute lung injury (ALI) and acute respiratory distress syndrome (ARDS) are associated with diverse disorders and characterized by disruption of the alveolar-capillary barrier, leakage of edema fluid into the lung, and substantial inflammation leading to acute respiratory failure. Gene therapy is a potentially powerful...

journal_title：Gene therapy

authors： Lin X,Barravecchia M,Kothari P,Young JL,Dean DA

更新日期：2016-06-01 00:00:00

abstract：:A retroviral vector constructed from the murine leukemia virus (MLV) can only express transgenes in cells undergoing mitosis, indicating its suitability as a delivery vehicle for cancer gene therapy. However, the transduction efficiency (TE) of retroviruses embedding endogenous envelope proteins in human cancer cells ...

journal_title：Gene therapy

authors： Lee H,Song JJ,Kim E,Yun CO,Choi J,Lee B,Kim J,Chang JW,Kim JH

更新日期：2001-02-01 00:00:00

abstract：:Introducing exogenous genes into cells is one of the most important molecular techniques to study gene functions. Comparing to other type of cells, neurons are more difficult to transfect with cDNAs because they are very sensitive to microenvironmental changes. Among various gene transfer techniques, the Ca(2+)-phosph...

journal_title：Gene therapy

authors： Jiang M,Deng L,Chen G

更新日期：2004-09-01 00:00:00

abstract：:betaTC-tet cells are conditionally immortalized pancreatic beta cells which can confer long-term correction of hyperglycemia when transplanted in syngeneic streptozocin diabetic mice. The use of these cells for control of type I diabetes in humans will require their encapsulation and transplantation in non-native site...

journal_title：Gene therapy

authors： Dupraz P,Rinsch C,Pralong WF,Rolland E,Zufferey R,Trono D,Thorens B

更新日期：1999-06-01 00:00:00

abstract：:Therapeutic RNA interference (RNAi) has emerged as a promising approach for the treatment of many incurable diseases, including cancer, infectious disease or neurodegenerative disorders. Demonstration of efficacy and safety in animal models is necessary before planning human application. Our group and others have prev...

journal_title：Gene therapy

authors： Martin JN,Wolken N,Brown T,Dauer WT,Ehrlich ME,Gonzalez-Alegre P

更新日期：2011-07-01 00:00:00

abstract：:Small interfering RNAs (siRNAs) are emerging as promising therapeutic tools. However, the widespread clinical application of such molecules as modulators of gene expression is still dependent on several aspects that limit their bioavailability. One of the most promising strategies to overcome the barriers faced by gen...

journal_title：Gene therapy

authors： Dharmapuri S,Peruzzi D,Marra E,Palombo F,Bett AJ,Bartz SR,Yong M,Ciliberto G,La Monica N,Buser CA,Toniatti C,Aurisicchio L

更新日期：2011-07-01 00:00:00

abstract：:Production of retroviral vectors for clinical use requires removal of cells and cellular debris. We combined a series of filters of decreasing pore size using commercially available blood banking filters approved for clinical use. The collection bag and filters can be connected to create a sterile, closed system using...

journal_title：Gene therapy

authors： Reeves L,Cornetta K

更新日期：2000-12-01 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