Evaluation of the biological differences of canine and human factor VIII in gene delivery: implications in human hemophilia treatment.

abstract：:Many retroviral vectors based on murine leukaemia virus (MLV) contain the first 420 nucleotides of the gag gene, as this was reported to increase vector titre by increasing the efficiency of RNA packaging. In this study, deletion of this gag sequence from its original location did not decrease the titre of two retrovi...

journal_title：Gene therapy

authors： Zhao Y,Low W,Collins MK

更新日期：2000-02-01 00:00:00

abstract：:Success of gene therapy for diseases affecting the T cell lineage depends on the thymic repopulation by genetically engineered hematopoietic progenitor cells (HPC). Although it has been shown that retrovirally transduced HPC can repopulate the thymus, little information is available on the effect of the culture protoc...

journal_title：Gene therapy

authors： Verhasselt B,Naessens E,De Smedt M,Plum J

更新日期：2000-05-01 00:00:00

abstract：:Genetic engineering of T cells for adoptive immunotherapy in cancer patients has shown significant promise. To ensure optimal antitumor activity and safety, the simultaneous expression of multiple genes is frequently required, and short viral-derived 2A sequences are increasingly preferred for this purpose. Concerns e...

journal_title：Gene therapy

authors： Arber C,Abhyankar H,Heslop HE,Brenner MK,Liu H,Dotti G,Savoldo B

更新日期：2013-09-01 00:00:00

abstract：:Sustainable correction of severe human genetic disorders of self-renewing tissues, such as the blistering skin disease junctional epidermolysis bullosa (JEB), is facilitated by stable genomic integration of therapeutic genes into somatic tissue stem cells. While integrating viral vectors can achieve this, they suffer ...

journal_title：Gene therapy

authors： Ortiz-Urda S,Lin Q,Yant SR,Keene D,Kay MA,Khavari PA

更新日期：2003-07-01 00:00:00

abstract：:The growth and metastasis of solid tumors relies on the activities of polypeptide growth factors to recruit stromal tissue and expand the tumor mass. Pleiotrophin (PTN) is a secreted growth factor with angiogenic activity that has been found to contribute to the growth and metastasis of tumors including melanoma. Here...

journal_title：Gene therapy

authors： Malerczyk C,Schulte AM,Czubayko F,Bellon L,Macejak D,Riegel AT,Wellstein A

更新日期：2005-02-01 00:00:00

abstract：:Adenovirus (Ad) vectors are of utility for many therapeutic applications. Strategies have been developed to alter adenoviral tropism to achieve a cell-specific gene delivery capacity employing fiber modifications allowing genetic incorporation of targeting motifs. In this regard, single chain antibodies (scFv) represe...

journal_title：Gene therapy

authors： Hedley SJ,Auf der Maur A,Hohn S,Escher D,Barberis A,Glasgow JN,Douglas JT,Korokhov N,Curiel DT

更新日期：2006-01-01 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：:Adeno-associated virus (AAV) derived vectors are considered highly eligible vehicles for human gene therapy. Not only do they possess many great potential for clinical applications due to their wide range of tissue targets but also their excellent preclinical safety profile makes them particularly suitable candidates ...

journal_title：Gene therapy

authors： Le Bec C,Douar AM

更新日期：2006-05-01 00:00:00

abstract：:The majority of immunotherapy-based gene therapy protocols consist of ex vivo gene transfer in tumor cells. To prevent further in vivo growth, modified cells must be irradiated before reinjection into patients. The present study examines the effects of gamma-irradiation on transgene expression in transduced leukemic c...

journal_title：Gene therapy

authors： Vereecque R,Saudemont A,Wickham TJ,Gonzalez R,Hetuin D,Fenaux P,Quesnel B

更新日期：2003-02-01 00:00:00

abstract：:Selection of targeted vectors from virus display peptide libraries is a versatile and efficient approach to improve vector specificity and efficiency. This strategy has been used to target various cell types in vitro. Here, we report the screening of an adeno-associated virus type 2 (AAV2) display peptide library in v...

journal_title：Gene therapy

authors： Ying Y,Müller OJ,Goehringer C,Leuchs B,Trepel M,Katus HA,Kleinschmidt JA

更新日期：2010-08-01 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：:Persistence of human immunodeficiency virus (HIV) despite highly active antiretroviral therapy (HAART) is a lasting challenge to virus eradication. To develop a strategy complementary to HAART, we constructed a series of Rev-dependent lentiviral vectors carrying diphtheria toxin A chain (DT-A) and its attenuated mutan...

journal_title：Gene therapy

authors： Wang Z,Tang Z,Zheng Y,Yu D,Spear M,Iyer SR,Bishop B,Wu Y

更新日期：2010-09-01 00:00:00

abstract：:The potential for gene therapy to be an effective treatment for cystic fibrosis has been hampered by the limited gene transfer efficiency of current vectors. We have shown that recombinant Sendai virus (SeV) is highly efficient in mediating gene transfer to differentiated airway epithelial cells, because of its capaci...

journal_title：Gene therapy

authors： Ferrari S,Griesenbach U,Iida A,Farley R,Wright AM,Zhu J,Munkonge FM,Smith SN,You J,Ban H,Inoue M,Chan M,Singh C,Verdon B,Argent BE,Wainwright B,Jeffery PK,Geddes DM,Porteous DJ,Hyde SC,Gray MA,Hasegawa M,Alton

更新日期：2007-10-01 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：:We demonstrated previously that the additive-type recombinant Sendai virus (rSeV) is highly efficient for use in pulmonary gene transfer; however, rSeV exhibits inflammatory responses. To overcome this problem, we tested newly developed non-transmissible constructs, namely, temperature-sensitive F-deleted vector, rSeV...

journal_title：Gene therapy

authors： Tanaka S,Yonemitsu Y,Yoshida K,Okano S,Kondo H,Inoue M,Hasegawa M,Masumoto K,Suita S,Taguchi T,Sueishi K

更新日期：2007-07-01 00:00:00

abstract：:Vectors conjugated with ligands recognized by cell surface receptors are of interest for cystic fibrosis gene therapy since these vectors would allow cell-specific targeting. However, an efficient and specific uptake may be abrogated by a subsequent intracellular trafficking leading to an inefficient gene transfer. Th...

journal_title：Gene therapy

authors： Fajac I,Grosse S,Briand P,Monsigny M

更新日期：2002-06-01 00:00:00

abstract：:Interferon-alpha (IFN-alpha) or CD80 transduction of tumor cells individually reduces tumorigenicity and enhances antitumor responses. Here, we report that the combination of IFN-alpha and CD80 cancer gene therapy in poorly immunogenic murine tumor models, the colorectal adenocarcinoma cell line MC38, and the methylch...

journal_title：Gene therapy

authors： Hiroishi K,Tüting T,Tahara H,Lotze MT

更新日期：1999-12-01 00:00:00

abstract：:Adeno-associated virus (AAV)-mediated gene delivery systems have been shown to be effective tools for gene manipulation in the inner ear. For example, hair cells (HCs) and multiple other cell types can be transduced by the local injection of AAVs into the inner ear. However, application of the AAV-mediated CRISPR/Cas9...

journal_title：Gene therapy

authors： Kang W,Zhao X,Sun Z,Dong T,Jin C,Tong L,Zhu W,Tao Y,Wu H

更新日期：2020-08-01 00:00:00

abstract：:Reactive oxygen species (ROS) have been implicated in the pathogenesis of rheumatoid arthritis (RA), while antioxidant enzymes, such as extracellular superoxide dismutase (EC-SOD) and catalase, block radical-induced events. The present study tested if the ex vivo transfer of EC-SOD and catalase genes alone or in combi...

journal_title：Gene therapy

authors： Dai L,Claxson A,Marklund SL,Feakins R,Yousaf N,Chernajovsky Y,Winyard PG

更新日期：2003-04-01 00:00:00

abstract：:Chimeric antigen receptors (CARs, immunoreceptors) are frequently used to redirect T cells with pre-defined specificity, in particular towards tumour cells for use in adoptive immunotherapy of malignant diseases. Specific targeting is mediated by an extracellularly located antibody-derived binding domain, which is joi...

journal_title：Gene therapy

authors： Hombach A,Hombach AA,Abken H

更新日期：2010-10-01 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：: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：:This study examines the clinical relevance of tissue engineering integrating gene therapy and polymer science to bone regeneration. Bilateral maxillary defects (3 x 1.2 cm(2)) in 20 miniature swine were bridged with a bioresorbable internal splint. Constructs were created using ex vivo adenovirus bone morphogenetic pr...

journal_title：Gene therapy

authors： Chang SC,Chuang HL,Chen YR,Chen JK,Chung HY,Lu YL,Lin HY,Tai CL,Lou J

更新日期：2003-11-01 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：: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：: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：:The clinical efficacy and safety as well as the application range of gene therapy will be broadened by developing systems capable of finely modulating the expression of therapeutic genes. Transgene regulation will be crucial for maintaining appropriate levels of a gene product within the therapeutic range, thus preven...

journal_title：Gene therapy

authors： Toniatti C,Bujard H,Cortese R,Ciliberto G

更新日期：2004-04-01 00:00:00

abstract：:Mobilized blood CD34+ cells from cancer patients were ex vivo infected by a recombinant adenovirus vector carrying an alkaline phosphatase gene, whose expression is evaluable by flow cytometry. A mean of 40% CD34+ cells were infected by the vector, with high levels of expression of the transgene. Among attempts to imp...

journal_title：Gene therapy

authors： Bregni M,Shammah S,Malaffo F,Di Nicola M,Milanesi M,Magni M,Matteucci P,Ravagnani F,Jordan CT,Siena S,Gianni AM

更新日期：1998-04-01 00:00:00

abstract：:We tested the longevity of gene expression provided by autonomously replicating vectors. The vectors contain segments of human genomic DNA that provide efficient replication initiation and sequences derived from Epstein-Barr virus that provide nuclear retention. In order to monitor gene expression, the vectors also ca...

journal_title：Gene therapy

authors： Wohlgemuth JG,Kang SH,Bulboaca GH,Nawotka KA,Calos MP

更新日期：1996-06-01 00:00:00

abstract：:First-generation adenoviral (Ad) vectors are frequently used vectors for experimental and clinical gene transfer. Earlier it has been shown that parallel overexpression of the cell cycle regulator p21(Waf1/Cip1) (p21) or antiapoptotic bcl-2 from a second vector reduces cytotoxicity and improves transgene expression. H...

journal_title：Gene therapy

authors： Schumacher A,Horvat S,Woischwill C,Wolff G,Witt C

更新日期：2009-04-01 00:00:00