EIAV vector-mediated delivery of endostatin or angiostatin inhibits angiogenesis and vascular hyperpermeability in experimental CNV.

abstract：:We recently showed that the human telomerase reverse transcriptase (hTERT) promoter induces tumor-specific Bax gene expression and selectively kills various human cancer cells both in vitro and in xenograft tumors. However, it remains unclear whether the hTERT promoter can be used to induce transgene expression in syn...

journal_title：Gene therapy

authors： Gu J,Andreeff M,Roth JA,Fang B

更新日期：2002-01-01 00:00:00

abstract：:Recombinant adeno-associated virus (rAAV) vectors are attractive candidates for the treatment of inherited and acquired retinal disease. Although rAAV vectors are well characterized in rodent models, a prerequisite to their clinical application in human patients is the thorough evaluation of their efficacy and safety ...

journal_title：Gene therapy

authors： Bainbridge JW,Mistry A,Schlichtenbrede FC,Smith A,Broderick C,De Alwis M,Georgiadis A,Taylor PM,Squires M,Sethi C,Charteris D,Thrasher AJ,Sargan D,Ali RR

更新日期：2003-08-01 00:00:00

abstract：:Viral gene vectors often rely on packaging cell lines, which provide the necessary factors in trans for the formation of virus-like particles. Previously, we reported on a first-generation packaging cell line for gene vectors, which are based on the B-lymphotropic Epstein-Barr virus (EBV), a human gamma-herpesvirus. T...

journal_title：Gene therapy

authors： Hettich E,Janz A,Zeidler R,Pich D,Hellebrand E,Weissflog B,Moosmann A,Hammerschmidt W

更新日期：2006-05-01 00:00:00

abstract：:Recombinant Semliki Forest virus (SFV) is an attractive viral vector system owing to its ability to allow high efficiency of viral protein expression. To produce recombinant pseudotyped human immunodeficiency virus type 1 (HIV-1) virions, we designed a chimeric SFV/HIV vector system that contains both the HIV-1 cis- a...

journal_title：Gene therapy

authors： Del Vecchio C,Calistri A,Lombardi G,Celegato M,Biasolo MA,Palù G,Parolin C

更新日期：2009-02-01 00:00:00

abstract：:Because periodontal ligament (PDL) cells are reported to contain progenitor or stem cell populations, they are considered a beneficial cell source for clinical periodontal regeneration. Both bone morphogenetic protein 4 (BMP4) and human telomerase reverse transcriptase (hTERT) have essential roles in the modulation of...

journal_title：Gene therapy

authors： Mi HW,Lee MC,Fu E,Chow LP,Lin CP

更新日期：2011-05-01 00:00:00

abstract：:Recently developed helper virus-free methods of herpes simplex virus (HSV) amplicon vector packaging provide stocks that are virtually devoid of the cytotoxic component normally associated with traditional helper virus-based packaging methods. These approaches involve cotransfection of amplicon plasmid DNA with either...

journal_title：Gene therapy

authors： Bowers WJ,Howard DF,Brooks AI,Halterman MW,Federoff HJ

更新日期：2001-01-01 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：:The development of lentiviral vectors that use Rev-independent mechanisms of nuclear export for their genomic RNA could facilitate the construction of novel anti-HIV vectors. We have improved the titers of Rev-independent lentiviral vectors having the SRV-1 CTE by mutating the major splice donor and acceptor sites pre...

journal_title：Gene therapy

authors： Mautino MR,Keiser N,Morgan RA

更新日期：2000-08-01 00:00:00

abstract：:A novel class of cationic hyperbranched polymers, containing branched oligoethylenimine (OEI 800 Da) as core, diacrylate esters as linkers and oligoamines as surface modification, was synthesized and evaluated regarding their structure-activity relationship as gene carriers. We show that pseudodendritic core character...

journal_title：Gene therapy

authors： Russ V,Elfberg H,Thoma C,Kloeckner J,Ogris M,Wagner E

更新日期：2008-01-01 00:00:00

abstract：:To induce RNA interference (RNAi), either small interfering RNAs (siRNAs) are directly introduced into the cell or short hairpin RNAs (shRNAs) are expressed from a DNA vector. At present, shRNAs are commonly synthesized by RNA polymerase III (Pol III) promoters of the H1 and U6 RNAs. In this study, we designed and cha...

journal_title：Gene therapy

authors： Choy EY,Kok KH,Tsao SW,Jin DY

更新日期：2008-02-01 00:00:00

abstract：:Substantial advances in our understanding of lentivirus lifecycles and their various constituent proteins have permitted the bioengineering of lentiviral vectors now considered safe enough for clinical trials for both lethal and non-lethal diseases. They possess distinct properties that make them particularly suitable...

journal_title：Gene therapy

authors： Balaggan KS,Ali RR

更新日期：2012-02-01 00:00:00

abstract：:We have developed a novel gene transfer drug, HIV-IT(V), for the treatment of HIV infection in humans. HIV-IT(V) is a retroviral vector encoding the HIV-1 IIIB env and rev genes and a neomycin resistance marker gene (neor). We have recently reported that HIV-IT(V) administered intramuscularly to male mice localizes pr...

journal_title：Gene therapy

authors： Kamantigue E,Edwards W 3rd,Chada S,Brumm D,Austin M,Irwin M,Mento S,Kowal K,Sajjadi N

更新日期：1996-02-01 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：:Cancer cachexia, characterized by anorexia, weight loss and progressive tissue wasting, has been postulated to be mediated by various cytokines. However, the precise mechanism of cachexia induction is not fully explained. We have developed synthetic double-stranded oligodeoxynucleotides (ODN) as 'decoy' cis-elements t...

journal_title：Gene therapy

authors： Kawamura I,Morishita R,Tomita N,Lacey E,Aketa M,Tsujimoto S,Manda T,Tomoi M,Kida I,Higaki J,Kaneda Y,Shimomura K,Ogihara T

更新日期：1999-01-01 00:00:00

abstract：:Cationic liposomes provide a means to introduce genes into cells both ex vivo and in vivo. In the past few years their use has been described in several tissues, e.g. lungs, liver, endothelium, brain. In this study we evaluated a commercially available poly-cationic liposome formulation in delivering a reporter gene i...

journal_title：Gene therapy

authors： Vitiello L,Chonn A,Wasserman JD,Duff C,Worton RG

更新日期：1996-05-01 00:00:00

abstract：:Antibodies have long been used in biomedical science as in vitro tools for the identification, purification and functional manipulation of target antigens; they have been exploited in vivo for diagnostic and therapeutic applications as well. Recent advances in antibody engineering have now allowed the genes encoding a...

journal_title：Gene therapy

authors： Marasco WA

更新日期：1997-01-01 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：: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：: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：:Mesenchymal stem cells (MSC) are a group of clonogenic cells present among the bone marrow stroma and capable of multilineage differentiation into mesoderm-type cells such as osteoblasts, adipocytes and chondrocytes. Due to their ease of isolation and their differentiation potential, MSC are being introduced into clin...

journal_title：Gene therapy

authors： Abdallah BM,Kassem M

更新日期：2008-01-01 00:00:00

abstract：:Intraperitoneal (i.p.) recurrence of cisplatin-refractory and p53 mutant ovarian cancer is a major clinical problem, despite surgery and chemotherapy. dl1520 (ONYX-015) is an E1B-55 kDa gene-deleted adenovirus engineered selectively to replicate in and destroy cancer cells lacking functional p53. However, a correlatio...

journal_title：Gene therapy

authors： Heise C,Ganly I,Kim YT,Sampson-Johannes A,Brown R,Kirn D

更新日期：2000-11-01 00:00:00

abstract：:Low levels of expression in haemopoietic cells of the DNA repair protein O6-alkylguanine-DNA alkyltransferase (A Tase), is associated with the dose-limiting sensitivity of these cells to the chemotherapeutic chloroethylating and related methylating agents. Thus, the use of agents which deplete ATase such as O6-benzylg...

journal_title：Gene therapy

authors： Hickson I,Fairbairn LJ,Chinnasamy N,Dexter TM,Margison GP,Rafferty JA

更新日期：1996-10-01 00:00:00

abstract：:Both atherosclerosis and arterial interventions induce oxidative stress mediated in part by nicotinamide adenine dinucleotide phosphate (NADPH) oxidases that have a pivotal role in the development of neointimal hyperplasia and restenosis. For small interfering RNA (siRNA) targeting of the NOX2 (Cybb) component of the ...

journal_title：Gene therapy

authors： Li JM,Newburger PE,Gounis MJ,Dargon P,Zhang X,Messina LM

更新日期：2010-10-01 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：: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：:In the last two decades, remarkable advances have been made in the development of technologies used to engineer new aptamers and ribozymes. This has encouraged interest among researchers who seek to create new types of gene-control systems that can be made to respond specifically to small-molecule signals. Validation ...

journal_title：Gene therapy

authors： Link KH,Breaker RR

更新日期：2009-10-01 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：:Human adenovirus (AV) is a favored vector for delivery of therapeutic genes into certain target cells, such as skeletal muscle cells for gene therapy. Here we show that replication-defective (E1 + E3 deleted) human type 5 adenovirus (AV) recombinants containing a reporter gene insert (RSV-luciferase or RSV-Lux) can ve...

journal_title：Gene therapy

authors： Acsadi G,Jani A,Huard J,Blaschuk K,Massie B,Holland P,Lochmüller H,Karpati G

更新日期：1994-09-01 00:00:00

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：: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