abstract：:Glioblastomas account for approximately 20% of all primary brain tumors in adults. Glioblastoma multiforme (GBM) is a highly malignant tumor. In spite of advances in surgery, chemotherapy, and radiotherapy, the life expectancy of the patient with glioblastoma is approximately 11 months. To enhance glioma-specific gene...

journal_title：Human gene therapy

authors： Ho IA,Lam PY,Hui KM

更新日期：2004-08-01 00:00:00

abstract：:The use of gene-engineered T cells expressing chimeric single-chain (scFv) receptors capable of codelivering CD28 costimulation and T cell receptor zeta chain (TCR-zeta) activation signals has emerged as a promising treatment regimen for cancer. Using retroviral transduction, primary human T lymphocytes were gene-engi...

journal_title：Human gene therapy

authors： Teng MW,Kershaw MH,Moeller M,Smyth MJ,Darcy PK

更新日期：2004-07-01 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：: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

abstract：:Targeted delivery of intravenously administered genetically altered cells or stem cells is still in an early stage of investigation. We developed a method of delivering iron oxide (ferumoxide)-labeled mesenchymal stem cells (MSCs) to a targeted area in an animal model by applying an external magnet. Rats with or witho...

journal_title：Human gene therapy

authors： Arbab AS,Jordan EK,Wilson LB,Yocum GT,Lewis BK,Frank JA

更新日期：2004-04-01 00:00:00

abstract：:Adenoviruses (Ads) have shown great utility as vectors for the delivery of genes to mammalian cells, partly because of their ability to infect a wide range of different cell types independent of the replicative state of the cell. However, Ads do not transduce mature muscle efficiently because of low levels of the natu...

journal_title：Human gene therapy

authors： Bramson JL,Grinshtein N,Meulenbroek RA,Lunde J,Kottachchi D,Lorimer IA,Jasmin BJ,Parks RJ

更新日期：2004-02-01 00:00:00

abstract：:Foamy virus (FV) vectors are a promising gene delivery system for use in hematopoietic stem cell gene therapy. Previous FV vector marking studies in the NOD/SCID xenotransplantation model used umbilical cord blood (UCB)-derived SCID repopulating cells (SRCs) that were assayed 5-10 weeks posttransplantation. We now rep...

journal_title：Human gene therapy

authors： Josephson NC,Trobridge G,Russell DW

更新日期：2004-01-01 00:00:00

abstract：:RNA interference (RNAi) is an evolutionarily conserved mechanism of posttranscriptional gene-specific silencing. For in vivo applications, RNAi has been hampered until recently by inefficient delivery methods and by the transient nature of the gene suppression. Lentiviral vectors (LVs) hold great promise for gene ther...

journal_title：Human gene therapy

authors： Van den Haute C,Eggermont K,Nuttin B,Debyser Z,Baekelandt V

更新日期：2003-12-10 00:00:00

abstract：:The optimal stem cell source for stem cell gene therapy has not been defined. Most gene transfer studies have used peripheral blood or marrow repopulating cells collected after administration of granulocyte colony-stimulating factor and stem cell factor (G-CSF/SCF). For clinical applications, however, growth factor ad...

journal_title：Human gene therapy

authors： Thomasson B,Peterson L,Thompson J,Goerner M,Kiem HP

更新日期：2003-11-20 00:00:00

abstract：:Interleukin (IL)-12 has been reported to induce cellular immune responses for protection against tumor formation. Here we investigate the utility of adenoviral delivery of IL-12 as an adjuvant for a human papillomavirus E7 subunit vaccine in a mouse tumor challenge model. Direct intratumoral injection of AdIL-12 resul...

journal_title：Human gene therapy

authors： Ahn WS,Bae SM,Kim TY,Kim TG,Lee JM,Namkoong SE,Kim CK,Sin JI

更新日期：2003-10-10 00:00:00

abstract：:Tumor angiogenesis is a rate-limiting factor for tumor growth, and the endothelial cells of tumor vessels display specific features that can be exploited for the selective delivery of cancer therapeutics. To specifically target exogenous genes to angiogenic tumor vessels, we generated a panel of vesicular stomatitis v...

journal_title：Human gene therapy

authors： De Palma M,Venneri MA,Naldini L

更新日期：2003-08-10 00:00:00

abstract：:MDA-MB-231, an HLA-A2(+), HER2/neu(+) allogeneic breast cancer cell line genetically modified to express the costimulatory molecule CD80 (B7-1), was used to vaccinate 30 women with previously treated stage IV breast cancer. Expression of CD80 conferred the ability to deliver a costimulatory signal and thereby improved...

journal_title：Human gene therapy

authors： Dols A,Smith JW 2nd,Meijer SL,Fox BA,Hu HM,Walker E,Rosenheim S,Moudgil T,Doran T,Wood W,Seligman M,Alvord WG,Schoof D,Urba WJ

更新日期：2003-07-20 00:00:00

abstract：:Transferring therapeutic genes into the nociceptive system, including dorsal root ganglia (DRGs) and the spinal cord, is potentially a powerful approach for the treatment of chronic pain in humans. Adeno-associated viral vectors (AAVs) are particularly useful in delivering foreign genes to targeted tissues because the...

journal_title：Human gene therapy

authors： Xu Y,Gu Y,Wu P,Li GW,Huang LY

更新日期：2003-06-10 00:00:00

abstract：:Adenoviral vectors used in gene therapy are predominantly derived from adenovirus serotype 5 (Ad5), which infects a broad range of cells. Ad5 cell entry involves interactions with the coxsackie-adenovirus receptor (CAR) and integrins. To assess these receptors in vivo, we mutated amino acid residues in fiber and pento...

journal_title：Human gene therapy

authors： Smith TA,Idamakanti N,Rollence ML,Marshall-Neff J,Kim J,Mulgrew K,Nemerow GR,Kaleko M,Stevenson SC

更新日期：2003-05-20 00:00:00

abstract：:In previous studies we demonstrated that a modified human HSP70b promoter (HSE.70b) directs high levels of gene expression to tumor cells after mild hyperthermia treatment in the range of 41.5-44 degrees C. This transcriptional targeting system exhibits low basal activity at 37 degrees C, is highly induced (950-fold) ...

journal_title：Human gene therapy

authors： Brade AM,Szmitko P,Ngo D,Liu FF,Klamut HJ

更新日期：2003-03-20 00:00:00

abstract：:Successful gene transfer into articular cartilage is a prerequisite for gene therapy of articular joint disorders. In the present study we tested the hypothesis that recombinant adeno-associated virus (rAAV) vectors are capable of effecting gene transfer in isolated articular chondrocytes in vitro, articular cartilage...

journal_title：Human gene therapy

authors： Madry H,Cucchiarini M,Terwilliger EF,Trippel SB

更新日期：2003-03-01 00:00:00

abstract：:Clinical applications of gene therapy require advances in gene delivery systems. Although numerous clinical trials are already underway, the ultimate success of gene therapies will depend on gene transfer vectors that facilitate the expression of a specific gene at therapeutic levels in the desired cell populations wi...

journal_title：Human gene therapy

authors： Anderson DM,Hall LL,Ayyalapu AR,Irion VR,Nantz MH,Hecker JG

更新日期：2003-02-10 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：:RNA interference (RNAi) is a form of posttranscriptional gene silencing mediated by short double-stranded RNA, known as small interfering RNA (siRNA). These siRNAs are capable of binding to a specific mRNA sequence and causing its degradation. The recent demonstration of a plasmid vector that directs siRNA synthesis i...

journal_title：Human gene therapy

authors： Abbas-Terki T,Blanco-Bose W,Déglon N,Pralong W,Aebischer P

更新日期：2002-12-10 00:00:00

abstract：:We have shown that adenovirus-mediated manipulation of apoptotic genes such as bax could be a therapeutic option for prostate cancer. Unfortunately, the response of experimental prostate tumors to a single therapeutic gene of the apoptotic pathway is short-lived, and most of these tumors relapse after a short period o...

journal_title：Human gene therapy

authors： Zhang Y,Yu J,Unni E,Shao TC,Nan B,Snabboon T,Kasper S,Andriani F,Denner L,Marcelli M

更新日期：2002-11-20 00:00:00

abstract：:Heme oxygenase 1 (HO-1) is an inducible enzyme that catalyzes heme to generate bilirubin, ferritin, and carbon monoxide. Because enhanced expression of HO-1 confers protection against many types of cell and tissue damage by modulating apoptotic cell death or cytokine expression profiles, we hypothesized that adenoviru...

journal_title：Human gene therapy

authors： Tsuburai T,Suzuki M,Nagashima Y,Suzuki S,Inoue S,Hasiba T,Ueda A,Ikehara K,Matsuse T,Ishigatsubo Y

更新日期：2002-11-01 00:00:00

abstract：:Systemic administration of adenoviral vectors leads to activation of innate and antigen-specific immunity. In an attempt to diminish T and B cell-specific immune responses to E1-deleted adenoviral vectors, capsid proteins were modified with various activated monomethoxypolyethylene glycols (MPEGs). The impact of this ...

journal_title：Human gene therapy

authors： Croyle MA,Chirmule N,Zhang Y,Wilson JM

更新日期：2002-10-10 00:00:00

abstract：:Guidelines for testing gene therapy products for ecotropic replication-competent retrovirus (Eco-RCR) have not been delineated as they have for amphotropic viruses. To evaluate biologic assays that can detect these viruses, we compared an S(+)/L(-) assay and a marker rescue assay designed specifically for Eco-RCR dete...

journal_title：Human gene therapy

authors： Reeves L,Duffy L,Koop S,Fyffe J,Cornetta K

更新日期：2002-09-20 00:00:00

abstract：:Introduction of a new vaccine requires choosing a delivery system that provides safe administration and the desired level of immunogenicity. The safety, tolerability, and immunogenicity of three monthly 2.5-mg doses of a PfCSP DNA vaccine were evaluated in healthy volunteers as administered intramuscularly (IM) by nee...

journal_title：Human gene therapy

authors： Epstein JE,Gorak EJ,Charoenvit Y,Wang R,Freydberg N,Osinowo O,Richie TL,Stoltz EL,Trespalacios F,Nerges J,Ng J,Fallarme-Majam V,Abot E,Goh L,Parker S,Kumar S,Hedstrom RC,Norman J,Stout R,Hoffman SL

更新日期：2002-09-01 00:00:00

abstract：:Dystrophic epidermolysis bullosa (DEB) comprises a family of inherited blistering skin disorders for which no corrective therapy currently exists. In the most severe form, the Hallopeau-Siemens subtype (RDEB-HS), the epidermal adhesion protein collagen VII is absent from the skin as a consequence of null mutations in ...

journal_title：Human gene therapy

authors： Mecklenbeck S,Compton SH,Mejía JE,Cervini R,Hovnanian A,Bruckner-Tuderman L,Barrandon Y

更新日期：2002-09-01 00:00:00

abstract：:Bag-1 exerts powerful antiapoptotic effects by binding and stabilizing Bcl-2 and interacting with the tumor necrosis factor receptor type I-induced death signal. We examined the effects of overexpression of Bag-1 by ex vivo adenoviral gene transfer on cold (4 degrees C for 24 hr) ischemia/reperfusion (I/R) injury of r...

journal_title：Human gene therapy

authors： Sawitzki B,Amersi F,Ritter T,Fisser M,Shen XD,Ke B,Busuttil R,Volk HD,Kupiec-Weglinski JW

更新日期：2002-08-10 00:00:00

abstract：:Systemic administration of currently manufactured viral stocks has not so far achieved sufficient circulating titers to allow therapeutic targeting of metastatic disease. This is due to low initial viral titers, immune inactivation, nonspecific adhesion, and loss of particles. One way to exploit the elegant molecular ...

journal_title：Human gene therapy

authors： Harrington K,Alvarez-Vallina L,Crittenden M,Gough M,Chong H,Diaz RM,Vassaux G,Lemoine N,Vile R

更新日期：2002-07-20 00:00:00

abstract：:Gene transfer of reporter genes may trigger immune responses against the heterologous protein resulting in shortening of gene expression and inflammation. We generated transgenic rats expressing the lacZ gene under the control of the human immunodeficiency virus type 1 (HIV-1) long-terminal repeat (LTR) (HIV-lacZ) to ...

journal_title：Human gene therapy

authors： Ménoret S,Aubert D,Tesson L,Braudeau C,Pichard V,Ferry N,Anegon I

更新日期：2002-07-20 00:00:00

abstract：:The immunogenicity of adenovirus vectors remains a major obstacle to their safe and efficacious use for gene therapy. In order to identify T-cell epitopes directly from adenoviruses, four viral protein sequences were screened for the well-characterized 9-mer HLA-A2 binding motif. Peripheral blood mononuclear cells (PB...

journal_title：Human gene therapy

authors： Olive M,Eisenlohr L,Flomenberg N,Hsu S,Flomenberg P

更新日期：2002-07-01 00:00:00

abstract：:Previous studies have documented that the skin can be used as a bioreactor to produce proteins for systemic release to treat diseases. A gene-switch system has been developed that allows regulated expression of therapeutic genes. To determine whether this system could be used in the skin, we developed a transgenic mou...

journal_title：Human gene therapy

authors： Cao T,Tsai SY,O'Malley BW,Wang XJ,Roop DR

更新日期：2002-06-10 00:00:00

abstract：:Activation of T cells is necessary for efficient retroviral-mediated gene transfer. In addition, if the population of infused cells is to be limited to transduced cells, a means of positive selection is required. We describe a clinical scale procedure for activation of donor T cells with anti-CD3/CD28 beads followed b...

journal_title：Human gene therapy

authors： Orchard PJ,Blazar BR,Burger S,Levine B,Basso L,Nelson DM,Gordon K,McIvor RS,Wagner JE,Miller JS

更新日期：2002-05-20 00:00:00

abstract：:Lentiviral vectors are promising tools for gene transfer into the central nervous system. We have characterized in detail transduction with human immunodeficiency virus type 1 (HIV-1)-derived vectors encoding enhanced green fluorescent protein (eGFP) in the adult mouse brain. Different brain regions such as the striat...

journal_title：Human gene therapy

authors： Baekelandt V,Claeys A,Eggermont K,Lauwers E,De Strooper B,Nuttin B,Debyser Z

更新日期：2002-05-01 00:00:00

abstract：:Point mutations in the dystrophin gene cause dystrophin deficiency and muscular dystrophy in the mdx mouse and a subset of patients with Duchenne muscular dystrophy. As an approach to gene therapy for muscular dystrophies due to point mutations, we have studied the ability of RNA-DNA chimeric oligonucleotides (chimera...

journal_title：Human gene therapy

authors： Bertoni C,Rando TA

更新日期：2002-04-10 00:00:00

abstract：:Gutted adenoviral (Ad) vectors have a greater cloning capacity and elicit less immune response than conventional Ad vectors. Unfortunately, clinical use of gutted vectors has been slowed by production difficulties, including low yield and a tendency for recombinant virus to emerge. These two problems are related, beca...

journal_title：Human gene therapy

authors： Hartigan-O'Connor D,Barjot C,Crawford R,Chamberlain JS

更新日期：2002-03-01 00:00:00

abstract：:Cell-based gene transfer using a stent platform would provide a significant advantage in terms of site-specific gene expression in the vasculature. The current study presents a novel stent design that allows stable in vivo transgene expression over a 4-week period in the vasculature. A mesh-stent coated with fibronect...

journal_title：Human gene therapy

authors： Panetta CJ,Miyauchi K,Berry D,Simari RD,Holmes DR,Schwartz RS,Caplice NM

更新日期：2002-02-10 00:00:00

abstract：:Myelosuppression is the main side effect of cancer chemotherapy. An improved rate of retroviral vector-mediated gene transfer to hematopoietic stem cells, shown in more recent clinical trials, has created the basis to test the concept of myeloprotective gene therapy. We transplanted clinical-scale human peripheral blo...

journal_title：Human gene therapy

authors： Schiedlmeier B,Schilz AJ,Kühlcke K,Laufs S,Baum C,Zeller WJ,Eckert HG,Fruehauf S

更新日期：2002-01-20 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：:Conditionally replicative adenovirus (CRAd) vectors are designed for specific oncolytic replication in tumor tissues with concomitant sparing of normal cells. As such, CRAds offer an unprecedented level of anticancer potential for malignancies that have been refractory to previous cancer gene therapy interventions. CR...

journal_title：Human gene therapy

authors： Li H,Haviv YS,Derdeyn CA,Lam J,Coolidge C,Hunter E,Curiel DT,Blackwell JL

更新日期：2001-12-10 00:00:00

abstract：:Glaucoma, a group of optic neuropathies, is the leading cause of irreversible blindness. Neuronal apoptosis in glaucoma is primarily associated with high intraocular pressure caused by chronically impaired outflow of aqueous humor through the trabecular meshwork, a reticulum of mitotically inactive endothelial-like ce...

journal_title：Human gene therapy

authors： Loewen N,Fautsch MP,Peretz M,Bahler CK,Cameron JD,Johnson DH,Poeschla EM

更新日期：2001-11-20 00:00:00

abstract：:Human hematopoietic stem cells (HSCs) are poorly transduced by vectors based on adenovirus serotype 5 (Ad5). This is primarily due to the paucity of the coxsackievirus-Ad receptor on these cells. In an attempt to change the tropism of Ad5, we constructed a series of chimeric E1-deleted Ad5 vectors in which the shaft a...

journal_title：Human gene therapy

authors： Knaän-Shanzer S,Van Der Velde I,Havenga MJ,Lemckert AA,De Vries AA,Valerio D

更新日期：2001-11-01 00:00:00