Single-cycle viral gene expression, rather than progressive replication and oncolysis, is required for VSV therapy of B16 melanoma.

abstract：:Successful keratinocyte gene therapy requires the development of efficient methods of gene transfer to keratinocytes. Jet injection of a solution containing DNA can be used to transfer genes to several tissues in vivo. In this article, we tried to introduce DNA into rat and human keratinocytes using this method. First...

journal_title：Gene therapy

authors： Sawamura D,Ina S,Itai K,Meng X,Kon A,Tamai K,Hanada K,Hashimoto I

更新日期：1999-10-01 00:00:00

abstract：:Fibrodysplasia ossificans progressiva (FOP) is an autosomal dominant congenital disorder characterized by progressive heterotopic bone formation. Currently, no definitive treatment exists for FOP. The activin receptor type IA / activin-like kinase 2 (ACVR1/ALK2) gene has been identified as the responsible gene for FOP...

journal_title：Gene therapy

authors： Takahashi M,Katagiri T,Furuya H,Hohjoh H

更新日期：2012-07-01 00:00:00

abstract：:As soon as RNA interference (RNAi) was found to work in mammalian cells, research quickly focused on harnessing this powerful endogenous and specific mechanism of gene silencing for human therapy. RNAi uses small RNAs, less than 30 nucleotides in length, to suppress expression of genes with complementary sequences. Tw...

journal_title：Gene therapy

authors： Dykxhoorn DM,Palliser D,Lieberman J

更新日期：2006-03-01 00:00:00

abstract：:Rescue of dystrophic skeletal muscle in mdx and utrophin/dystrophin-deficient (dko) mouse models by reintroduction of dystrophin has validated gene therapy as a potential therapeutic approach for Duchenne muscular dystrophy. However, the size of the dystrophin gene exceeds the capacity of adeno-associated viral (AAV) ...

journal_title：Gene therapy

authors： Gardner KL,Kearney JA,Edwards JD,Rafael-Fortney JA

更新日期：2006-05-01 00:00:00

abstract：:The possibility of achieving multiple systemic expression of human interferon-beta in mice upon repeated intravenous administration of cationic liposome-DNA complex (lipoplex) was investigated. Lipoplexes containing the pentammonio lipid pcTG90 were first optimized by selecting the most efficient ratio of pcTG90 to ph...

journal_title：Gene therapy

authors： Meyer O,Schughart K,Pavirani A,Kolbe HV

更新日期：2000-09-01 00:00:00

abstract：:We introduce an injectable system for the formation of a biodegradable DNA-containing implant that releases DNA over a 2-month period to provide a robust and prolonged gene expression at the site. Sustained delivery of the appropriate plasmid DNA resulted in sustained expression of luciferase, the persistent appearanc...

journal_title：Gene therapy

authors： Eliaz RE,Szoka FC Jr

更新日期：2002-09-01 00:00:00

abstract：:Development of efficient short-term gene transfer technologies for embryonic stem (ES) cells is urgently needed for various existing and new ES cell-based research strategies. In this study, we present a highly efficient, nonviral non-DNA technology for genetic loading of mouse ES cells based on electroporation of def...

journal_title：Gene therapy

authors： Ponsaerts P,Brown JP,Van den Plas D,Van den Eeden L,Van Bockstaele DR,Jorens PG,Van Tendeloo VF,Merregaert J,Singh PB,Berneman ZN

更新日期：2004-11-01 00:00:00

abstract：:A replication-incompetent adenoviral vector encoding the heavy chain C-fragment (H(C)50) of botulinum neurotoxin type C (BoNT/C) was evaluated as a mucosal vaccine against botulism in a mouse model. Single intranasal inoculation of the adenoviral vector elicited a high level of H(C)50-specific IgG, IgG1 and IgG2a in s...

journal_title：Gene therapy

authors： Xu Q,Pichichero ME,Simpson LL,Elias M,Smith LA,Zeng M

更新日期：2009-03-01 00:00:00

abstract：:In an earlier study exploring the potential of gene transfer to repair myocardial conduction defects, we observed that myotubes, generated by forced expression of MyoD, exhibit reduced excitability when also modified to express connexin43 (Cx43). We hypothesized that this effect was caused by gap junction-mediated cou...

journal_title：Gene therapy

authors： Kizana E,Ginn SL,Smyth CM,Boyd A,Thomas SP,Allen DG,Ross DL,Alexander IE

更新日期：2006-11-01 00:00:00

abstract：:We created Na(+)/HCO3(-) cotransporter 1 (NBCe1) p.W516* knock-in mice as a model of isolated proximal renal tubular acidosis showing early lethality associated with severe metabolic acidosis to investigate the therapeutic effects of prenatal alkalization or posttranscriptional control 124 (PTC124). NBCe1(W516*/W516*)...

journal_title：Gene therapy

authors： Fang YW,Yang SS,Chau T,Nakamura M,Yamazaki O,Seki G,Yamada H,Hsu HM,Cheng CJ,Lin SH

更新日期：2015-05-01 00:00:00

abstract：:Aptamers are oligonucleotides evolved in vitro or in nature to bind target ligands with high affinity and specificity. They are emerging as powerful tools in the fields of therapeutics, drug development, target validation and diagnostics. Aptamers are attractive alternatives to antibody- and small-molecule-based thera...

journal_title：Gene therapy

authors： Que-Gewirth NS,Sullenger BA

更新日期：2007-02-01 00:00:00

abstract：:Recombinant Autographa californica multiple nuclear polyhedrosis viruses (AcMNPV) have recently been shown to transduce mammalian cells in vitro. Since baculoviruses offer many advantages over viruses currently used in gene therapy, we have tested them for in vivo gene transfer by constructing a baculovirus bearing a ...

journal_title：Gene therapy

authors： Airenne KJ,Hiltunen MO,Turunen MP,Turunen AM,Laitinen OH,Kulomaa MS,Ylä-Herttuala S

更新日期：2000-09-01 00:00:00

abstract：:Retroviral vectors are effective shuttle systems by introducing therapeutically relevant genes stably into the genome of proliferating cells. The majority of vectors applied for research or clinical applications use neomycin for cell selection and identification. To circumvent the time consuming and potentially toxic ...

journal_title：Gene therapy

authors： Machl AW,Planitzer S,Kubbies M

更新日期：1997-04-01 00:00:00

abstract：:To facilitate the understanding of the complex process of target gene expression and its control, we report a modified inducible system for activation or repression of target gene expression in response to an exogenously administered compound. The main component of this inducible system is a chimeric transcriptional a...

journal_title：Gene therapy

authors： Wang Y,Xu J,Pierson T,O'Malley BW,Tsai SY

更新日期：1997-05-01 00:00:00

abstract：:The efficiency of plasmid gene transfer to skeletal muscle can be significantly improved by the application of an electrical field to the muscle following injection of plasmid DNA. However, this electrotransfer is associated with significant muscle damage which may result in substantial loss of transfected muscle fibr...

journal_title：Gene therapy

authors： McMahon JM,Signori E,Wells KE,Fazio VM,Wells DJ

更新日期：2001-08-01 00:00:00

abstract：:The E1 deleted adenoviral vectors are efficient at gene transfer to cells in culture or in animals. However, their use is limited because of an immune-mediated loss of transduced cells. This immune response is believed to result from low-level production of viral antigens from these vectors after gene transfer. The ea...

journal_title：Gene therapy

authors： Scaria A,Curiel DT,Kay MA

更新日期：1995-06-01 00:00:00

abstract：:Active disease of tuberculosis (TB) can be developed decades later by either a relapse of the initial infection (endogenous reactivation) or by an entrance of the secondary infection (exogenous reinfection), since the current chemotherapy cannot lead to complete elimination of tuberculosis. Although the immunotherapeu...

journal_title：Gene therapy

authors： Ha SJ,Jeon BY,Youn JI,Kim SC,Cho SN,Sung YC

更新日期：2005-04-01 00:00:00

abstract：:Homologous restriction factor 20 (HRF20, CD59) is one of the complement regulatory factors. In this study, the complement-resistant retroviral vector, which possesses the HRF20 gene as a selection gene, was constructed and examined. The virus-producing cell, transduced with complement-resistant retroviral vector, was ...

journal_title：Gene therapy

authors： Hayashi S,Emi N,Okada H,Nagasaka T,Yokoyama I,Takagi H

更新日期：1998-02-01 00:00:00

abstract：:As clinical gene therapy has progressed toward realizing its potential, concern over misuse of the technology to enhance performance in athletes is growing. Although 'gene doping' is banned by the World Anti-Doping Agency, its detection remains a major challenge. In this study, we developed a methodology for direct de...

journal_title：Gene therapy

authors： Baoutina A,Coldham T,Bains GS,Emslie KR

更新日期：2010-08-01 00:00:00

abstract：:The annual rate of kidney graft loss caused by chronic allograft nephropathy (CAN) has not improved over the past decade. Recent reports suggest that acute renal ischemia results in development of CAN. The goal of the present study was to assess the renoprotective potential and safety of hepatocyte growth factor (HGF)...

journal_title：Gene therapy

authors： Isaka Y,Yamada K,Takabatake Y,Mizui M,Miura-Tsujie M,Ichimaru N,Yazawa K,Utsugi R,Okuyama A,Hori M,Imai E,Takahara S

更新日期：2005-05-01 00:00:00

abstract：:Adenovirus vectors have great potential in cancer gene therapy. Targeting of cancer-testis (CT) antigens, which are specifically presented at the surface of tumor cells by human leukocyte antigen (HLA) class I molecules, is an attractive option. In this study, a single-chain T-cell receptor (scTCR) directed against th...

journal_title：Gene therapy

authors： de Vrij J,Uil TG,van den Hengel SK,Cramer SJ,Koppers-Lalic D,Verweij MC,Wiertz EJ,Vellinga J,Willemsen RA,Hoeben RC

更新日期：2008-07-01 00:00:00

abstract：:Highly active antiretroviral therapy (HAART) treatment for HIV has changed the course of AIDS in societies in which the drugs are readily available. Despite the great success of HAART, drug resistance and toxicity issues still remain a concern for some individuals. Thus, a number of investigators have been exploring o...

journal_title：Gene therapy

authors： Zhou J,Rossi JJ

更新日期：2011-12-01 00:00:00

abstract：:Selection of targeted vectors from virus display peptide libraries is a versatile and efficient approach to improve vector specificity and efficiency. This strategy has been used to target various cell types in vitro. Here, we report the screening of an adeno-associated virus type 2 (AAV2) display peptide library in v...

journal_title：Gene therapy

authors： Ying Y,Müller OJ,Goehringer C,Leuchs B,Trepel M,Katus HA,Kleinschmidt JA

更新日期：2010-08-01 00:00:00

abstract：:Vectors conjugated with ligands recognized by cell surface receptors are of interest for cystic fibrosis gene therapy since these vectors would allow cell-specific targeting. However, an efficient and specific uptake may be abrogated by a subsequent intracellular trafficking leading to an inefficient gene transfer. Th...

journal_title：Gene therapy

authors： Fajac I,Grosse S,Briand P,Monsigny M

更新日期：2002-06-01 00:00:00

abstract：:Inadequate antiprotease activity in the lungs due to alpha-1-antitrypsin (A1AT) deficiency is a factor of early-onset emphysema. We propose a new approach to gene therapy that involves the intratracheal delivery of macrophages expressing human A1AT (hA1AT). Recombinant adeno-associated virus (rAAV) plasmids encoding t...

journal_title：Gene therapy

authors： Zhang D,Wu M,Nelson DE,Pasula R,Martin WJ 2nd

更新日期：2003-12-01 00:00:00

abstract：:Dendritic cell (DC)-based vaccine approaches are being actively evaluated for developing immunotherapeutic agents against cancers. In this study, we investigated the use of engineered DCs expressing transgenic tumor-associated antigen hgp100 and the regulatory cytokine interleukin-21, namely DC-hgp100/mIL-21, as a the...

journal_title：Gene therapy

authors： Aravindaram K,Wang PH,Yin SY,Yang NS

更新日期：2014-05-01 00:00:00

abstract：:Until recently, adeno-associated virus 9 (AAV9) was considered the AAV serotype most effective in crossing the blood-brain barrier (BBB) and transducing cells of the central nervous system (CNS), following systemic injection. However, a newly engineered capsid, AAV-PHP.B, is reported to cross the BBB at even higher ef...

journal_title：Gene therapy

authors： Rincon MY,de Vin F,Duqué SI,Fripont S,Castaldo SA,Bouhuijzen-Wenger J,Holt MG

更新日期：2018-04-01 00:00:00

abstract：:Efficient transduction of the peripheral nervous system (PNS) is required for gene therapy of acquired and inherited neuropathies, neuromuscular diseases and for pain treatment. We have characterized the tropism and transduction efficiency of different adeno-associated vectors (AAV) pseudotypes after sciatic nerve inj...

journal_title：Gene therapy

authors： Homs J,Ariza L,Pagès G,Udina E,Navarro X,Chillón M,Bosch A

更新日期：2011-06-01 00:00:00

abstract：:In muscle, mutant genes can be targeted and corrected directly by intramuscular (i.m.) injection of corrective DNA, or by ex vivo delivery of DNA to myogenic cells, followed by cell transplantation. Short fragment homologous replacement (SFHR) has been used to repair the exon 23 nonsense transition at the Xp21.1 dys l...

journal_title：Gene therapy

authors： Kapsa RM,Quigley AF,Vadolas J,Steeper K,Ioannou PA,Byrne E,Kornberg AJ

更新日期：2002-06-01 00:00:00

abstract：:The clinical efficacy and safety as well as the application range of gene therapy will be broadened by developing systems capable of finely modulating the expression of therapeutic genes. Transgene regulation will be crucial for maintaining appropriate levels of a gene product within the therapeutic range, thus preven...

journal_title：Gene therapy

authors： Toniatti C,Bujard H,Cortese R,Ciliberto G

更新日期：2004-04-01 00:00:00