Pulmonary inflammation induced by incomplete or inactivated adenoviral particles.

abstract：:DNA-based vaccines able to induce efficient cytotoxic T-cell responses targeting conserved elements (CE) of human immunodeficiency virus type 1 (HIV-1) Gag have been developed. These CE were selected by stringent conservation, the ability to induce T-cell responses with broad human leukocyte antigen coverage, and the ...

journal_title：Human gene therapy

authors： Hu X,Valentin A,Cai Y,Dayton F,Rosati M,Ramírez-Salazar EG,Kulkarni V,Broderick KE,Sardesai NY,Wyatt LS,Earl PL,Moss B,Mullins JI,Pavlakis GN,Felber BK

更新日期：2018-09-01 00:00:00

abstract：:Classical gene therapy for cystic fibrosis has had limited success because of immune response against viral vectors and short-term expression of cDNA-based transgenes. These limitations could be overcome by delivering the complete genomic CFTR gene on nonintegrating human artificial chromosomes (HACs). Here, we report...

journal_title：Human gene therapy

authors： Rocchi L,Braz C,Cattani S,Ramalho A,Christan S,Edlinger M,Ascenzioni F,Laner A,Kraner S,Amaral M,Schindelhauer D

更新日期：2010-09-01 00:00:00

abstract：:Restoration of correct splicing of βIVS2-654-globin pre-mRNA was previously accomplished in erythroid cells from β-thalassemia/HbE patients by an engineered U7 small nuclear RNA (snRNA) that carried a sequence targeted to the cryptic branch point and an exonic splicing enhancer, U7.BP+623 snRNA. In this study, this ap...

journal_title：Human gene therapy

authors： d'Arqom A,Nualkaew T,Jearawiriyapaisarn N,Kole R,Svasti S

更新日期：2020-11-02 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：:Stem cell mobilization to injured tissue contributes to neovascularization, resulting in regeneration after myocardial infarction (MI). We previously showed that direct cardiac injection of a recombinant lentivirus (LV) that engineers expression of membrane-bound stem cell factor (mSCF) improves outcomes immediately a...

journal_title：Human gene therapy

authors： Sun Z,Lee CJ,Mejia-Guerrero S,Zhang Y,Higuchi K,Li RK,Medin JA

更新日期：2012-12-01 00:00:00

abstract：:The T cell co-stimulatory molecule B7-1 was transduced into a poorly immunogenic murine neuroblastoma cell line (Neuro-2a, N-2a) alone or in combination with MHC class II genes to test the ability of these genes to stimulate antitumor immunity. N-2a cells transduced with B7-1 exhibited reduced tumorigenicity, whereas ...

journal_title：Human gene therapy

authors： Heuer JG,Tucker-McClung C,Gonin R,Hock RA

更新日期：1996-11-10 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：:Gene-modified replication-competent adenoviruses (Ads) are emerging as a promising new modality for the treatment of cancer. We have previously shown that E1B 19kDa and E1B 55kDa gene-deleted Ad (Ad-DeltaE1B19/55) exhibits improved tumor-specific replication and cell lysis, leading to an enhanced antitumor effect. In ...

journal_title：Human gene therapy

authors： Kim J,Kim JH,Choi KJ,Kim PH,Yun CO

更新日期：2007-09-01 00:00:00

abstract：:Lentiviral vectors hold great promise for the genetic correction of various inherited diseases. However, lentiviral vector biology is still not completely understood and warrants the precise decoding of molecular mechanisms underlying integration and post-translational modification. This study investigated a series of...

journal_title：Human gene therapy

authors： Scholz SJ,Fronza R,Bartholomä CC,Cesana D,Montini E,von Kalle C,Gil-Farina I,Schmidt M

更新日期：2017-10-01 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：:Defined serum-free conditions have great conceptual advantages for the biological safety and standardization of clinical gene transfer into hematopoietic stem cells. In the only study reported to date, Sekhar et al. achieved low serum conditions by a complex concentration procedure of a retroviral supernatant initiall...

journal_title：Human gene therapy

authors： Glimm H,Flügge K,Möbest D,Hofmann VM,Postmus J,Henschler R,Lange W,Finke J,Kiem HP,Schulz G,Rosenthal F,Mertelsmann R,von Kalle C

更新日期：1998-04-10 00:00:00

abstract：:Three-vessel disease (TVD) is a severe coronary heart disease (CHD) with poor prognosis. Niemann-Pick C1-like 1 (NPC1L1) is a transporter protein for exogenous cholesterol absorption, and 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) is a rate-limiting enzyme for cholesterol synthesis. We aimed to investigat...

journal_title：Human gene therapy

authors： Zhao X,Li J,Tang X,Liu R,Xu J,Xu L,Jiang L,Huang K,Tian J,Feng X,Wu Y,Zhang Y,Wang D,Sun K,Xu B,Zhao W,Hui R,Gao R,Song L,Yuan J

更新日期：2021-01-22 00:00:00

abstract：:The utility of first-generation adenovirus vectors for long-term gene transfer in humans is limited by preexisting antiadenoviral immunity. We demonstrate here that new-generation high-capacity adenovirus vectors (HC-Ads) can efficiently transduce the brain and mediate stable transgene expression for at least 2 months...

journal_title：Human gene therapy

authors： Thomas CE,Schiedner G,Kochanek S,Castro MG,Lowenstein PR

更新日期：2001-05-01 00:00:00

abstract：:CTL lines directed against HIV-1 antigens were generated from infected individuals and were transduced by the HMB-K(b)HuIFNbeta vector, resulting in low, constitutive expression of interferon beta (IFN-beta). The IFN-beta-transduced cells showed markedly increased HIV-1-specific, MHC class I-restricted CTL activity ag...

journal_title：Human gene therapy

authors： Hadida F,De Maeyer E,Cremer I,Autran B,Baggiolini M,Debré P,Vieillard V

更新日期：1999-07-20 00:00:00

abstract：:The inherited deficiency in adenosine deaminase (ADA), which results in severe combined immunodeficiency, is generally regarded as an optimal model for the development of human somatic gene therapy. The ideal target for the correction of ADA deficiency and other lympho-hematopoietic disorders would be the hematopoieti...

journal_title：Human gene therapy

authors： Cournoyer D,Scarpa M,Mitani K,Moore KA,Markowitz D,Bank A,Belmont JW,Caskey CT

更新日期：1991-10-01 00:00:00

abstract：:Doxycycline (DOX) is widely used as a pharmacological agent and as an effector molecule in inducible gene expression systems. For most applications, it is important to determine whether the DOX concentration reaches the level required for optimal efficacy. We developed a sensitive bioassay for measuring the DOX concen...

journal_title：Human gene therapy

authors： Kleibeuker W,Zhou X,Centlivre M,Legrand N,Page M,Almond N,Berkhout B,Das AT

更新日期：2009-05-01 00:00:00

abstract：:Adenovirus-polylysine-DNA complexes were evaluated for their capacity to accomplish direct in vivo gene transfer to airway epithelium employing a rodent model. Binary complexes containing transferrin or adenovirus, or combination complexes containing both transferrin and adenovirus, were evaluated. The highest in vitr...

journal_title：Human gene therapy

authors： Gao L,Wagner E,Cotten M,Agarwal S,Harris C,Rømer M,Miller L,Hu PC,Curiel D

更新日期：1993-02-01 00:00:00

abstract：:Newcastle disease virus (NDV) is a naturally oncolytic virus that has been shown to be safe and effective for cancer therapy. Tumor virotherapy using NDV emerged in the 1950s and has advanced more recently by the increased availability of reverse genetics technology. In this study, we constructed a reverse genetics sy...

journal_title：Human gene therapy

authors： Wei D,Sun N,Nan G,Wang Y,Liu HQ,Peeters B,Chen ZN,Bian H

更新日期：2012-07-01 00:00:00

abstract：:Production of clinical-grade gammaretroviral vectors for ex vivo gene delivery requires a scalable process that can rapidly generate large amounts of vector supernatant, clear large numbers of residual packaging cells with minimal decreases in vector titer, and satisfy all current regulatory guidelines regarding produ...

journal_title：Human gene therapy

authors： Feldman SA,Goff SL,Xu H,Black MA,Kochenderfer JN,Johnson LA,Yang JC,Wang Q,Parkhurst MR,Cross S,Morgan RA,Cornetta K,Rosenberg SA

更新日期：2011-01-01 00:00:00

abstract：:The influence of serum on the production of retroviral vectors by the HT1080 human fibrosarcoma-derived packaging cell line FLYRD18 was investigated. A fourfold increase in virus titer was observed under serum-free conditions, as compared with medium supplemented with 10% fetal calf serum. A similar improvement was al...

journal_title：Human gene therapy

authors： Gerin PA,Gilligan MG,Searle PF,Al-Rubeai M

更新日期：1999-08-10 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：:Recombinant adeno-associated virus 2 (rAAV2) has been extensively used as a gene delivery vector for the nervous system. It targets primarily neurons in the nervous system and results in sustained long-term expression of transgenes. New rAAV serotypes have been characterized and demonstrated to have improved transduct...

journal_title：Human gene therapy

authors： Burger C,Nash K,Mandel RJ

更新日期：2005-07-01 00:00:00

abstract：:This multicenter phase I/II study evaluated the safety, pharmacokinetics, and antitumor effects of repeated doses of NV1020, a genetically engineered oncolytic herpes simplex virus, in patients with advanced metastatic colorectal cancer (mCRC). Patients with liver-dominant mCRC received four fixed NV1020 doses via wee...

journal_title：Human gene therapy

authors： Geevarghese SK,Geller DA,de Haan HA,Hörer M,Knoll AE,Mescheder A,Nemunaitis J,Reid TR,Sze DY,Tanabe KK,Tawfik H

更新日期：2010-09-01 00:00:00

abstract：:The hepatocarcinoma-intestine-pancreas (HIP) gene, also called pancreatitis-associated protein-1 (PAP1) or Reg IIIalpha, is activated in most human hepatocellular carcinomas (HCCs) but not in normal liver, which suggests that HIP regulatory sequence could be used as efficient liver tumor-specific promoters to express ...

journal_title：Human gene therapy

authors： Hervé J,Cunha AS,Liu B,Valogne Y,Longuet M,Boisgard R,Brégerie O,Roux J,Guettier C,Calès P,Tavitian B,Samuel D,Clerc J,Bréchot C,Faivre J

更新日期：2008-09-01 00:00:00

abstract：:To achieve effective gene therapy, it is necessary to selectively and efficiently transfect therapeutic gene into targeted cells. In this study, we developed a combination method using mannosylated lipoplexes, which show selectivity to antigen-presenting cells such as macrophages and dendritic cells, and bubble liposo...

journal_title：Human gene therapy

authors： Un K,Kawakami S,Suzuki R,Maruyama K,Yamashita F,Hashida M

更新日期：2010-01-01 00:00:00

abstract：:Despite efforts toward improvements in retrovirus-mediated gene transfer, stable high-level expression of a therapeutic gene in human hematopoietic stem cells remains a great challenge. We have evaluated the efficiency of different viral long terminal repeats (LTRs) in long-term expression of a transgene in vivo, usin...

journal_title：Human gene therapy

authors： Kaneko S,Onodera M,Fujiki Y,Nagasawa T,Nakauchi H

更新日期：2001-01-01 00:00:00

abstract：:Bone marrow mesenchymal stem cells (BMSCs) represent an important source of cells for tissue repair. The tropism of these cells to the sites of injury and tumors has been well established. Their tumor-homing properties make BMSCs good candidates as antitumor agent delivery vehicles. In this study, we showed that BMSCs...

journal_title：Human gene therapy

authors： Song C,Xiang J,Tang J,Hirst DG,Zhou J,Chan KM,Li G

更新日期：2011-04-01 00:00:00

abstract：:The efficiency of gene therapy strategies against cancer is limited by the poor distribution of the vectors in the malignant tissues. To solve this problem, a new generation of tumor-specific, conditionally replicative adenoviruses is being developed. To direct the replication of the virus to breast cancer, we have co...

journal_title：Human gene therapy

authors： Hernandez-Alcoceba R,Pihalja M,Wicha MS,Clarke MF

更新日期：2000-09-20 00:00:00

abstract：:Overexpression of human manganese-containing superoxide dismutase (MnSOD) activity has been demonstrated to suppress malignancy in human melanoma and breast carcinoma cells in vitro and in vivo. To study its effects on human oral squamous carcinoma cells, stable transfection and expression of MnSOD in SCC-25 cells hav...

journal_title：Human gene therapy

authors： Liu R,Oberley TD,Oberley LW

更新日期：1997-03-20 00:00:00

abstract：:Direct arterial gene transfer has been previously achieved using double-balloon catheters and perforated balloons, in most cases facilitated by the use of cationic liposomes or viral vectors. These gene delivery systems, however, have been compromised by issues relating to efficacy and/or safety, and furthermore requi...

journal_title：Human gene therapy

authors： Riessen R,Rahimizadeh H,Blessing E,Takeshita S,Barry JJ,Isner JM

更新日期：1993-12-01 00:00:00