Capsid Engineering of Adenovirus Vectors: Overcoming Early Vector-Host Interactions for Therapy.

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：: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：: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：:We report on an antitumor treatment involving electrogene therapy (EGT), a newly developed in vivo gene transfer method using electroporation. We carried out in vivo EGT in a subcutaneous model of CT26 colon carcinoma cells, using plasmid DNAs encoding interleukin 12 (IL-12) subunits. For this purpose, we developed tw...

journal_title：Human gene therapy

authors： Tamura T,Nishi T,Goto T,Takeshima H,Dev SB,Ushio Y,Sakata T

更新日期：2001-07-01 00:00:00

abstract：:The rapid inactivation of murine-derived retroviral vectors in human or nonhuman primate sera is largely attributed to the activity of complement mediated through the classical pathway. In this study, we have further investigated the relationship between the human complement cascade and retrovirus inactivation. Preinc...

journal_title：Human gene therapy

authors： Rother RP,Squinto SP,Mason JM,Rollins SA

更新日期：1995-04-01 00:00:00

abstract：:This study was designed to retrovirally transduce T cells by a protocol that would be simple, short, cost effective, applicable for clinical use, and efficient enough to avoid further selection of transduced T cells. Because retrovirally mediated infection is depending on the cell cycle, we first optimized the conditi...

journal_title：Human gene therapy

authors： Movassagh M,Boyer O,Burland MC,Leclercq V,Klatzmann D,Lemoine FM

更新日期：2000-05-20 00:00:00

abstract：:We employed the hypophysectomized rats as an animal model to explore the feasibility of using genetically engineered fibroblast cells for growth hormone gene therapy. An internal ribosome entry site (IRES)-directed bicistronic retroviral vector, PSN, which contained a porcine growth hormone (pGH) cDNA at the first cis...

journal_title：Human gene therapy

authors： Chen BF,Chang WC,Chen ST,Chen DS,Hwang LH

更新日期：1995-07-01 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：: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：:Pancreatic cancer is the fourth leading cause of cancer-related death in the United States, and even under optimal therapy these patients face a poor prognosis. Here we report a novel gene therapy-based strategy to battle this disease. We show that the majority of pancreatic tumors overexpress c-erb-B2, which therefor...

journal_title：Human gene therapy

authors： Liu X,Li J,Tian Y,Xu P,Chen X,Xie K,Qiu Z,Wang Y,Zhang D,Wolf F,Li C,Huang Q

更新日期：2010-02-01 00:00:00

abstract：:Fabry disease, caused by a deficiency of lysosomal enzyme alpha-galactosidase A (alpha-gal A), is one of the inherited disorders potentially treatable by gene transfer to hematopoietic stem cells. In this study, a high-titer amphotropic retroviral producer cell line, MFG-alpha-gal A, was established. CD34+ cells from ...

journal_title：Human gene therapy

authors： Takiyama N,Dunigan JT,Vallor MJ,Kase R,Sakuraba H,Barranger JA

更新日期：1999-12-10 00:00:00

abstract：:Subcutaneous vaccination therapy with glioma cells, which are retrovirally transduced to secrete granulocyte-macrophage colony-stimulating factor (GM-CSF), has previously proven effective in C57BL/6 mice harboring intracerebral GL261 gliomas. However, clinical ex vivo gene therapy for human gliomas would be difficult,...

journal_title：Human gene therapy

authors： Herrlinger U,Jacobs A,Quinones A,Woiciechowsky C,Sena-Esteves M,Rainov NG,Fraefel C,Breakefield XO

更新日期：2000-07-01 00:00:00

abstract：:Malignant pleural mesothelioma (MPM) is a fatal disease with a median survival of less than 14 months. For the first time, a genetically engineered vaccinia virus is shown to produce efficient infection, replication, and oncolytic effect against MPM. GLV-1h68 is a replication-competent engineered vaccinia virus carryi...

journal_title：Human gene therapy

authors： Kelly KJ,Woo Y,Brader P,Yu Z,Riedl C,Lin SF,Chen N,Yu YA,Rusch VW,Szalay AA,Fong Y

更新日期：2008-08-01 00:00:00

abstract：:Replication-deficient adenovirus vector (Ad) is one of the most efficient gene transfer vehicles for human gene therapy. However, Ad is antigenic, known to evoke prominent inflammatory responses in vivo, and there are concerns that using Ad in patients with immune-mediated disorders (allergy and autoimmune diseases) m...

journal_title：Human gene therapy

authors： Suzuki M,Suzuki S,Yamamoto N,Komatsu S,Inoue S,Hashiba T,Nishikawa M,Ishigatsubo Y

更新日期：2000-04-10 00:00:00

abstract：:Deficiencies in different steps of purine metabolism give rise to a number of human inherited disorders. Lesch-Nyhan syndrome is a severe neurological disorder, caused by a deficiency in the purine salvage enzyme hypoxanthine phosphoribosyltransferase (HPRT). HPRT-deficient mice have been generated, but have proved to...

journal_title：Human gene therapy

authors： Redhead NJ,Selfridge J,Wu CL,Melton DW

更新日期：1996-08-20 00:00:00

abstract：:Development of multidrug resistance (MDR) is the major obstacle to successful cancer chemotherapy. We have developed Daudi human lymphoma cells that are 20-fold more resistant than the parent cell line to vincristine (VCR) by infecting cells with pHaMDR1/A retroviral vector (Daudi/MDR20). Three DNA sequences of anti-M...

journal_title：Human gene therapy

authors： Wang FS,Kobayashi H,Liang KW,Holland JF,Ohnuma T

更新日期：1999-05-01 00:00:00

abstract：:The gene therapy strategy using the hsvl-thymidine kinase gene (TK) and ganciclovir (GCV) injections that has been used for treating human glioblastomas has not been as effective as expected after the first animal experiments. A better understanding of the different steps involved in this treatment, like gene transfer...

journal_title：Human gene therapy

authors： Sturtz FG,Waddell K,Shulok J,Chen X,Caruso M,Sanson M,Snodgrass HR,Platika D

更新日期：1997-11-01 00:00:00

abstract：:Sphingosine kinase 1 (SPK1) has been identified as a central mediator of ischemia preconditioning and plays a protective role in ischemia/reperfusion (I/R)-induced cardiomyocyte death. In the present study, we investigated the protective effect of adenovirus-mediated SPK1 gene (Ad-SPK1) transfer on I/R-induced cardiac...

journal_title：Human gene therapy

authors： Duan HF,Wang H,Yi J,Liu HJ,Zhang QW,Li LB,Zhang T,Lu Y,Wu CT,Wang LS

更新日期：2007-11-01 00:00:00

abstract：:Gene therapy for Duchenne muscular dystrophy will likely require that the corrective dystrophin gene be delivered to a high fraction of muscle fibers in vivo. Because of the large size of the dystrophin cDNA, adenoviral (Ad) vectors have been developed for this application. However, Ad vectors transduce mature muscle ...

journal_title：Human gene therapy

authors： Menezes KM,Mok HS,Barry MA

更新日期：2006-03-01 00:00:00

abstract：:Cell therapies are treatments in which stem or progenitor cells are stimulated to differentiate into specialized cells able to home to and repair damaged tissues. After their discovery, endothelial progenitor cells (EPCs) stimulated worldwide interest as possible vehicles to perform autologous cell therapy of tumors. ...

journal_title：Human gene therapy

authors： Laurenzana A,Margheri F,Chillà A,Biagioni A,Margheri G,Calorini L,Fibbi G,Del Rosso M

更新日期：2016-10-01 00:00:00

abstract：:Glycogen storage disease type II (GSD-II) is a lethal, autosomal recessive metabolic myopathy caused by a lack of acid-alpha-glucosidase (GAA) activity in the cardiac and skeletal muscles. Absence of adequate intralysosomal GAA activity results in massive amounts of glycogen accumulation in multiple muscle groups, res...

journal_title：Human gene therapy

authors： Ding EY,Hodges BL,Hu H,McVie-Wylie AJ,Serra D,Migone FK,Pressley D,Chen YT,Amalfitano A

更新日期：2001-05-20 00:00:00

abstract：:First-generation adenoviral (Ad) and high-capacity adenoviral (HC-Ad) vectors are efficient delivery vehicles for transferring therapeutic transgenes in vivo into tissues/organs. The initial successes reported with adenoviral vectors in preclinical trials have been limited by immune-related adverse side effects. This ...

journal_title：Human gene therapy

authors： Puntel M,Curtin JF,Zirger JM,Muhammad AK,Xiong W,Liu C,Hu J,Kroeger KM,Czer P,Sciascia S,Mondkar S,Lowenstein PR,Castro MG

更新日期：2006-05-01 00:00:00

abstract：:Recombinant vectors based on adeno-associated virus serotype 8 (AAV8) have been successfully used in the clinic and hold great promise for liver-directed gene therapy. Preexisting immunity against AAV8 or the development of antibodies against the therapeutic transgene product might negatively affect the outcomes of ge...

journal_title：Human gene therapy

authors： Ferla R,Claudiani P,Savarese M,Kozarsky K,Parini R,Scarpa M,Donati MA,Sorge G,Hopwood JJ,Parenti G,Fecarotta S,Nigro V,Sivri HS,Van Der Ploeg A,Andria G,Brunetti-Pierri N,Auricchio A

更新日期：2015-03-01 00:00:00

abstract：:Hepatic stellate cells (HSCs) are the primary cell type responsible for liver fibrogenesis. Transforming growth factor beta 1 (TGF-β1) and platelet-derived growth factor (PDGF) are key profibrotic cytokines that regulate HSC activation and proliferation with functional convergence. Dual RNA interference against their ...

journal_title：Human gene therapy

authors： Jiang Y,Zhao Y,He F,Wang H

更新日期：2019-02-01 00:00:00

abstract：:Human immunodeficiency virus (HIV) infection represents one of the most challenging systems for gene therapy. Thanks to the extended knowledge of the molecular biology of the HIV life cycle, many different strategies have been developed including transdominant modifications of HIV proteins, RNA decoys, antisense RNA, ...

journal_title：Human gene therapy

authors： Michienzi A,Conti L,Varano B,Prislei S,Gessani S,Bozzoni I

更新日期：1998-03-20 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：:Naked plasmid DNA electrotransfer offers advantages over viral-based gene delivery, including being regulatory permissive, but factors influencing expression efficiency and cell fate impact on translational utility. This study compared co-expression of red and green fluorescence reporter plasmids with differing promot...

journal_title：Human gene therapy

authors： Pinyon JL,Klugmann M,Lovell NH,Housley GD

更新日期：2019-02-01 00:00:00

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：:Administration of plasmid/lipid complexes to the lung airways for the treatment of metastatic pulmonary diseases represents a new strategy of gene therapy. In this study we present evidence that intratracheal administration of a plasmid encoding murine IL-12 complexed with N-[1-(2,3-dioleyloxy)propyl)-N,N,N-trimethyla...

journal_title：Human gene therapy

authors： Blezinger P,Freimark BD,Matar M,Wilson E,Singhal A,Min W,Nordstrom JL,Pericle F

更新日期：1999-03-20 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