Helper virus-free herpes simplex virus type 1 amplicon vectors for granulocyte-macrophage colony-stimulating factor-enhanced vaccination therapy for experimental glioma.

abstract：:A major obstacle for the efficacy of cancer gene therapy is the need to transduce a high proportion of tumor cells with genes that directly or indirectly cause their death. During the formation of certain organs, cells compete among themselves to colonize the whole tissue. We reasoned that cell competition could be us...

journal_title：Human gene therapy

authors： Martinez-Quintanilla J,Cascallo M,Fillat C,Alemany R

更新日期：2009-07-01 00:00:00

abstract：:Replication-deficient adenoviruses are known to induce acute injury and inflammation of infected tissues, thus limiting their use for human gene therapy. However, molecular mechanisms triggering this response have not been fully defined. To characterize this response, chemokine expression was evaluated in DBA/2 mice f...

journal_title：Human gene therapy

authors： Muruve DA,Barnes MJ,Stillman IE,Libermann TA

更新日期：1999-04-10 00:00:00

abstract：:Three retroviral constructs containing a full-length human alpha-L-iduronidase (IDUA) cDNA were made. The first, pLIdSN, is designed so that expression of the IDUA cDNA is from the 5' viral long terminal repeat (LTR). The second, pLNCId, is designed to express the IDUA cDNA from the cytomegalovirus (CMV) immediate ear...

journal_title：Human gene therapy

authors： Anson DS,Bielicki J,Hopwood JJ

更新日期：1992-08-01 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：:Cotransfer of a therapeutic gene together with the human MDR1 gene provides an opportunity to increase the number of transduced marrow cells, expressing the therapeutic gene, by in vivo selection for MDR1. We have used an Lg-MDR1-IRES-neo (LgMIN) retroviral vector, containing MDR1 and neo genes, separated by the EMCV ...

journal_title：Human gene therapy

authors： Zaboikin MM,Schuening FG

更新日期：1998-10-10 00:00:00

abstract：:Immune cells are involved in the pathogenesis of osteoarthritis (OA). CD4(+) T cells were activated during the onset of OA and induced macrophage inflammatory protein (MIP)-1γ expression and subsequent osteoclast formation. We evaluated the effects of local knockdown of MIP-1γ in a mouse OA model induced by anterior c...

journal_title：Human gene therapy

authors： Shen PC,Lu CS,Shiau AL,Lee CH,Jou IM,Hsieh JL

更新日期：2013-10-01 00:00:00

abstract：:Gene therapy has evolved into a tempting strategy for the management of cancer and other life-threatening diseases. Various approaches employ retroviral vectors to deliver the therapeutic gene. The profound knowledge about retrovirus biology allows the generation of increasingly advanced vector systems as well as an a...

journal_title：Human gene therapy

authors： Brandtner EM,Kodajova P,Knapp E,Ertl R,Tabotta W,Salmons B,Günzburg WH,Hohenadl C

更新日期：2008-01-01 00:00:00

abstract：:Although replication-deficient adenoviruses can efficiently transfer genes to the salivary glands, the current vectors precipitate an immediate, transient decrease in salivary function. To study the cause of this salivary hypofunction, 10(6)-10(10) plaque-forming units (pfu) of the vector AdCMV beta gal were delivered...

journal_title：Human gene therapy

authors： Adesanya MR,Redman RS,Baum BJ,O'Connell BC

更新日期：1996-06-10 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：:As an alternative to virus-mediated gene transfer, we previously demonstrated a simple, safe, and efficient transfer of foreign gene into the central nervous system using continuous injection of a plasmid DNA-cationic liposome complex. To explore whether this approach can be applied to the treatment of certain neurolo...

journal_title：Human gene therapy

authors： Imaoka T,Date I,Ohmoto T,Nagatsu T

更新日期：1998-05-01 00:00:00

abstract：:Antitumor gene therapy using herpes simplex type 1 thymidine kinase (TKh) and ganciclovir (GCV) treatment has revealed an important intratumoral bystander effect. A whole tumor can be eliminated when only a fraction of its tumor cells express TKh. We now report that the bystander effect not only acts within a tumor, b...

journal_title：Human gene therapy

authors： Kianmanesh AR,Perrin H,Panis Y,Fabre M,Nagy HJ,Houssin D,Klatzmann D

更新日期：1997-10-10 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：:Malignant mesothelioma is a tumor of the pleura for which there is no satisfactory treatment. It is almost universally fatal, regardless of the stage of the tumor at the time of diagnosis. Current treatment modalities include surgery, chemotherapy, and radiation therapy, although in some series none of these modalitie...

journal_title：Human gene therapy

authors： Schwarzenberger P,Harrison L,Weinacker A,Marrogi A,Byrne P,Ramesh R,Theodossiou C,Gaumer R,Summer W,Freeman SM,Kolls JK

更新日期：1998-11-20 00:00:00

abstract：:The central nervous system (CNS) is a predominant site of involvement in several lysosomal storage diseases (LSDs); and for many patients, these diseases are diagnosed only after the onset of symptoms related to the progressive accumulation of macromolecules within lysosomes. The mucopolysaccharidosis type VII (MPS VI...

journal_title：Human gene therapy

authors： Sferra TJ,Qu G,McNeely D,Rennard R,Clark KR,Lo WD,Johnson PR

更新日期：2000-03-01 00:00:00

abstract：:The use of synthetic gene delivery systems in human gene transfer is hampered by poor transfection efficiencies, largely because of the inability of DNA to translocate across the nuclear pore complex. A means to overcome this barrier is to bind the DNA to nuclear localization signals (NLSs), which are recognized by sh...

journal_title：Human gene therapy

authors： Mesika A,Kiss V,Brumfeld V,Ghosh G,Reich Z

更新日期：2005-02-01 00:00:00

abstract：:Three dogs with deficiency of the lysosomal enzyme alpha-L-iduronidase were treated by gene replacement therapy targeted at muscle. Direct intramuscular injections of plasmid encoding the alpha-L-iduronidase gene cDNA resulted in no detectable enzyme production, but may have resulted in immunologic sensitization to id...

journal_title：Human gene therapy

authors： Shull RM,Lu X,McEntee MF,Bright RM,Pepper KA,Kohn DB

更新日期：1996-08-20 00:00:00

abstract：:As efficient and less toxic virus-derived gene therapy vectors are developed, a pressing problem is to avoid immune response to the therapeutic gene product. Secreted therapeutic proteins potentially represent a special problem, as they are readily available to professional antigen-presenting cells throughout the body...

journal_title：Human gene therapy

authors： Cerullo V,McCormack W,Seiler M,Mane V,Cela R,Clarke C,Rodgers JR,Lee B

更新日期：2007-12-01 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：:Aberrant JAK/STAT3 pathway has been reported to be related to hepatocellular carcinoma (HCC) in many cell lines. In this study, a double-regulated oncolytic adenovirus vector that can replicate and induce a cytopathic effect in alpha-fetoprotein (AFP)-positive HCC cell lines with p53 dysfunction was successfully const...

journal_title：Human gene therapy

authors： Wei RC,Cao X,Gui JH,Zhou XM,Zhong D,Yan QL,Huang WD,Qian QJ,Zhao FL,Liu XY

更新日期：2011-09-01 00:00:00

abstract：:Recombinant adeno-associated virus 2 (rAAV) vectors have been successfully used for sustained expression of therapeutic genes. The potential of using rAAV as a cancer vaccine vector and the impact of a bacterial plasmid adjuvant on this activity were investigated. C57BL/6 mice received a single intramuscular injection...

journal_title：Human gene therapy

authors： Ponnazhagan S,Mahendra G,Lima J,Aldrich WA,Jenkins CB,Ren C,Kumar S,Kallman L,Strong TV,Shaw DR,Triozzi PL

更新日期：2004-09-01 00:00:00

abstract：:An alternative form of gene therapy involves immunoisolation of a nonautologous cell line engineered to secrete a therapeutic product. Encapsulation of these cells in a biocompatible polymer serves to protect these allogeneic cells from host-versus-graft rejection while recombinant products and nutrients are able to p...

journal_title：Human gene therapy

authors： Cirone P,Bourgeois JM,Shen F,Chang PL

更新日期：2004-10-01 00:00:00

abstract：:Efficient and homogeneous gene transfer to cardiac myocytes is a major target in myocardial gene therapy. The aim of this study was to determine the conditions permitting efficient, homogeneous, adenovirus-mediated gene transfer to cardiac myocytes, with a view to application during coronary artery catheterization. Ge...

journal_title：Human gene therapy

authors： Logeart D,Hatem SN,Rücker-Martin C,Chossat N,Névo N,Haddada H,Heimburger M,Perricaudet M,Mercadier JJ

更新日期：2000-05-01 00:00:00

abstract：:Adoptive cellular therapy provides the promise of a potentially powerful general treatment for cancer. Although this is a complex and challenging field, there have been major advances in basic and translational research resulting in clinical trial activity that is now beginning to confirm this promise. However, these ...

journal_title：Human gene therapy

authors： Hawkins RE,Gilham DE,Debets R,Eshhar Z,Taylor N,Abken H,Schumacher TN,ATTACK Consortium

更新日期：2010-06-01 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：:Mucopolysaccharidosis type IIIA (MPSIIIA) is a rare lysosomal storage disorder caused by mutations in the sulfamidase gene. Accumulation of glycosaminoglycan (GAG) inside the lysosomes is associated with severe neurodegeneration as well as peripheral organ pathological changes leading to death of affected individuals ...

journal_title：Human gene therapy

authors： Ruzo A,Marcó S,García M,Villacampa P,Ribera A,Ayuso E,Maggioni L,Mingozzi F,Haurigot V,Bosch F

更新日期：2012-12-01 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：:Immunoisolation of allogeneic cells within a membrane-bound device is a unique approach for gene therapy. We employed an immunoisolation device that protects allograft, but not xenograft, cells from destruction, to implant a human fibroblast line (MSU 1.2) in athymic rodents. Cells, transduced with the MFG-human facto...

journal_title：Human gene therapy

authors： Brauker J,Frost GH,Dwarki V,Nijjar T,Chin R,Carr-Brendel V,Jasunas C,Hodgett D,Stone W,Cohen LK,Johnson RC

更新日期：1998-04-10 00:00:00

abstract：:Although recombinant adeno-associated virus serotype 8 (AAV8) and serotype 5 (AAV5) vectors have shown efficacy in Phase 1 clinical trials for gene therapy of hemophilia B, it has become increasingly clear that these serotypes are not optimal for transducing primary human hepatocytes. We have previously reported that ...

journal_title：Human gene therapy

authors： Brown HC,Doering CB,Herzog RW,Ling C,Markusic DM,Spencer HT,Srivastava A,Srivastava A

更新日期：2020-10-01 00:00:00

abstract：:Deficiency of glycogen branching enzyme (GBE) causes glycogen storage disease type IV (GSD IV), which is characterized by the accumulation of a less branched, poorly soluble form of glycogen called polyglucosan (PG) in multiple tissues. This study evaluates the efficacy of gene therapy with an adeno-associated viral (...

journal_title：Human gene therapy

authors： Yi H,Zhang Q,Brooks ED,Yang C,Thurberg BL,Kishnani PS,Sun B

更新日期：2017-03-01 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