CDK4 and cyclin D1 allow human myogenic cells to recapture growth property without compromising differentiation potential.

abstract：:Gene delivery of angiogenic growth factors is a promising approach for the treatment of ischemic cardiovascular diseases. However, success of this new therapeutic principle is hindered by the lack of critical understanding as to how disease pathology affects the efficiency of gene delivery and/or the downstream signal...

journal_title：Gene therapy

authors： Qian HS,Liu P,Huw LY,Orme A,Halks-Miller M,Hill SM,Jin F,Kretschmer P,Blasko E,Cashion L,Szymanski P,Vergona R,Harkins R,Yu J,Sessa WC,Dole WP,Rubanyi GM,Kauser K

更新日期：2006-09-01 00:00:00

abstract：:More than one hundred different mutations in the gene encoding rhodopsin are associated with a group of retinal degenerations including retinitis pigmentosa, congenital stationary night blindness and retinitis punctata albescens. Given this large heterogeneity of mutations, it would be ideal to develop mutation-indepe...

journal_title：Gene therapy

authors： Cashman SM,Binkley EA,Kumar-Singh R

更新日期：2005-08-01 00:00:00

abstract：:We have used a particular folate receptor, which is overexpressed in tumor cells, for targeted DNA delivery into these cell types. This folate receptor internalizes folate through caveolae by a process named potocytosis, which is distinct from endocytosis, through clathrin-coated pits. When folate conjugated to poly-L...

journal_title：Gene therapy

authors： Gottschalk S,Cristiano RJ,Smith LC,Woo SL

更新日期：1994-05-01 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：:In 1998, two distinct and exciting scientific fields emerged which have profoundly shaped the current direction of biomedical research. The discovery of RNA interference (RNAi) and the derivation of human embryonic stem (ES) cells have yielded exciting new possibilities for researchers and clinicians alike. While fund...

journal_title：Gene therapy

authors： Heidersbach A,Gaspar-Maia A,McManus MT,Ramalho-Santos M

更新日期：2006-03-01 00:00:00

abstract：:Interferon-alpha (IFN-alpha) or CD80 transduction of tumor cells individually reduces tumorigenicity and enhances antitumor responses. Here, we report that the combination of IFN-alpha and CD80 cancer gene therapy in poorly immunogenic murine tumor models, the colorectal adenocarcinoma cell line MC38, and the methylch...

journal_title：Gene therapy

authors： Hiroishi K,Tüting T,Tahara H,Lotze MT

更新日期：1999-12-01 00:00:00

abstract：:By-pass surgery and percutaneous transluminal (coronary) angioplasty, PT(C)A, are standard techniques for the treatment of vascular occlusions. Their usefulness is limited by by-pass graft failure and restenosis occurring after the procedures. Twenty percent of patients treated with PTCA/PTA need a new revascularizati...

journal_title：Gene therapy

authors： Rutanen J,Puhakka H,Ylä-Herttuala S

更新日期：2002-11-01 00:00:00

abstract：:To achieve high transgene expression in the liver, we have compared the reporter gene expression among various murine retroviral long terminal repeats (LTRs) or leader sequences in vitro. Transient reporter gene expression assays revealed the highest gene expression by the polycythemic strain of spleen focus-forming v...

journal_title：Gene therapy

authors： Ohnishi N,Itoh K,Itoh Y,Baum C,Higashitsuji H,Yamaguchi K,Tsuji T,Okanoue T,Fujita J

更新日期：2002-02-01 00:00:00

abstract：:Regulated expression of therapeutic genes is required for long-term gene therapy applications for many disorders. Here we describe a doxycycline (dox)-regulated lentiviral vector system consisting of two HIV-1-based self-inactivating viruses. One of the vectors is constitutively expressing a novel improved version of ...

journal_title：Gene therapy

authors： Koponen JK,Kankkonen H,Kannasto J,Wirth T,Hillen W,Bujard H,Ylä-Herttuala S

更新日期：2003-03-01 00:00:00

abstract：:Several of the current techniques for transfer of both oligonucleotide and plasmid DNA into the myocardium are impaired by low efficiency and toxicity. To improve gene transfer techniques, especially into the whole heart, a gene transfer method involving liposome in conjunction with a viral envelope (HVJ-liposome) was...

journal_title：Gene therapy

authors： Sawa Y,Kaneda Y,Bai HZ,Suzuki K,Fujimoto J,Morishita R,Matsuda H

更新日期：1998-11-01 00:00:00

abstract：:Parkinson's disease is a prevalent progressive degenerative disorder of the elderly. There is a current need for novel therapeutic strategies because the standard levodopa pharmacotherapy is only temporarily efficacious. Recently, there have been some high-profile successful preclinical results obtained in animal mode...

journal_title：Gene therapy

authors： Manfredsson FP,Lewin AS,Mandel RJ

更新日期：2006-03-01 00:00:00

abstract：:Antibodies have long been used in biomedical science as in vitro tools for the identification, purification and functional manipulation of target antigens; they have been exploited in vivo for diagnostic and therapeutic applications as well. Recent advances in antibody engineering have now allowed the genes encoding a...

journal_title：Gene therapy

authors： Marasco WA

更新日期：1997-01-01 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：:To induce RNA interference (RNAi), either small interfering RNAs (siRNAs) are directly introduced into the cell or short hairpin RNAs (shRNAs) are expressed from a DNA vector. At present, shRNAs are commonly synthesized by RNA polymerase III (Pol III) promoters of the H1 and U6 RNAs. In this study, we designed and cha...

journal_title：Gene therapy

authors： Choy EY,Kok KH,Tsao SW,Jin DY

更新日期：2008-02-01 00:00:00

abstract：:Rhesus macaque tripartite motif (TRIM)5alpha potently inhibits early stages of human immunodeficiency virus (HIV)-1 replication, while the human orthologue has little effect on this virus. We used PCR-based random mutagenesis to construct a large library of human TRIM5alpha variants containing mutations in the PRYSPRY...

journal_title：Gene therapy

authors： Pham QT,Bouchard A,Grütter MG,Berthoux L

更新日期：2010-07-01 00:00:00

abstract：:Glucagon-like peptide 1 (GLP-1) and its analogue exendin-4 (Ex4) have displayed potent glucose homeostasis-modulating characteristics in type 2 diabetes (T2D). However, there are few reports of effectiveness in type 1 diabetes (T1D) therapy, where there is massive loss of beta cells. We previously described a novel GL...

journal_title：Gene therapy

authors： Soltani N,Kumar M,Glinka Y,Prud'homme GJ,Wang Q

更新日期：2007-06-01 00:00:00

abstract：:Retroviral vectors are being used increasingly in clinical gene therapy protocols but low transduction frequencies are presenting a significant obstacle to progress. In this paper we report a simple method to enhance the efficiency of ex vivo retroviral gene transfer. Calcium chloride is added to the vector stock and ...

journal_title：Gene therapy

authors： Morling FJ,Russell SJ

更新日期：1995-09-01 00:00:00

abstract：:Cultured keratinocyte allografts from unrelated donors can be readily grown as sheets in large-scale cell culture and have been used as an immediate skin cover for severely burned patients. Despite the absence of passenger leukocytes and the unlimited amount of material that can be obtained for permanent skin coverage...

journal_title：Gene therapy

authors： Mhashilkar AM,Doebis C,Seifert M,Busch A,Zani C,Soo Hoo J,Nagy M,Ritter T,Volk HD,Marasco WA

更新日期：2002-03-01 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：:The tumor-suppressor gene PTEN encodes a multifunctional phosphatase that is mutated in a variety of human cancers. PTEN inhibits the phosphatidylinositol 3-kinase pathway and downstream functions, including activation of Akt/protein kinase B (PKB), cell survival, and cell proliferation in tumor cells carrying mutant-...

journal_title：Gene therapy

authors： Saito Y,Swanson X,Mhashilkar AM,Oida Y,Schrock R,Branch CD,Chada S,Zumstein L,Ramesh R

更新日期：2003-11-01 00:00:00

abstract：:Cationic lipid-based delivery systems such as lipoplexes or stabilized plasmid-lipid particles (SPLP) represent a safer alternative to viral systems for gene therapy applications. We studied the impact of cell cycle status on the efficiency of transfection of human ovarian carcinoma tumor cells using two cationic-lipi...

journal_title：Gene therapy

authors： Mortimer I,Tam P,MacLachlan I,Graham RW,Saravolac EG,Joshi PB

更新日期：1999-03-01 00:00:00

abstract：:Oligonucleotides (ODNs) show great promise in their ability to specifically inhibit single gene expression but must cross the cell membrane, escape the endosomal vesicle, and possibly traverse the nuclear membrane to arrive at their intracellular target molecules. In an attempt to improve the delivery of phosphodieste...

journal_title：Gene therapy

authors： Ebbinghaus SW,Vigneswaran N,Miller CR,Chee-Awai RA,Mayfield CA,Curiel DT,Miller DM

更新日期：1996-04-01 00:00:00

abstract：:A number of genetic disorders are manifested in cutaneous epithelium and gene therapy approaches for treatment of such diseases are being considered. A successful gene therapy protocol requires durable and correctly targeted gene expression within the tissue. The continuous renewal and high levels of compartmentalizat...

journal_title：Gene therapy

authors： Ghazizadeh S,Doumeng C,Taichman LB

更新日期：2002-10-01 00:00:00

abstract：:Therapeutic RNA interference (RNAi) has emerged as a promising approach for the treatment of many incurable diseases, including cancer, infectious disease or neurodegenerative disorders. Demonstration of efficacy and safety in animal models is necessary before planning human application. Our group and others have prev...

journal_title：Gene therapy

authors： Martin JN,Wolken N,Brown T,Dauer WT,Ehrlich ME,Gonzalez-Alegre P

更新日期：2011-07-01 00:00:00

abstract：:Overexpression of vascular endothelial growth factor (VEGF) and its cognate receptor KDR has been linked to a more aggressive phenotype of human prostate carcinomas. The importance of signal transduction through the VEGF receptor 2 is illustrated by use of soluble KDR, which binds to VEGF and sequesters this ligand be...

journal_title：Gene therapy

authors： Kaliberov SA,Kaliberova LN,Buchsbaum DJ

更新日期：2005-03-01 00:00:00

abstract：:p53 gene therapy is being tested clinically for the treatment of human cancer, however, some cancer models (in vivo and in vitro) are resistant to p53. To explore the potential use of two p53 homologues, p73 and p51/p63, in cancer gene therapy, we introduced p53, p73 and p51/p63 into colorectal cancer cell lines via a...

journal_title：Gene therapy

authors： Sasaki Y,Morimoto I,Ishida S,Yamashita T,Imai K,Tokino T

更新日期：2001-09-01 00:00:00

abstract：:We constructed a melanoma-specific oncolytic adenoviral vector Ad.MCDIRESE1.71Hsp3, in which the cytosine deaminase and adenoviral E1A genes linked by the IRES sequence were under the control of a mouse tyrosinase enhancer/promoter transcriptional element in the E1 region of the vector. We also inserted the human heat...

journal_title：Gene therapy

authors： Liu Y,Ye T,Sun D,Maynard J,Deisseroth A

更新日期：2006-08-01 00:00:00

abstract：:In-stent restenosis results exclusively from neointimal hyperplasia due to mechanical injury and a foreign body response to the prosthesis. Inflammation mediated by monocyte chemoattractant protein-1 (MCP-1) might therefore underlie in-stent restenosis. We recently devised a new strategy for anti-MCP-1 gene therapy by...

journal_title：Gene therapy

authors： Ohtani K,Usui M,Nakano K,Kohjimoto Y,Kitajima S,Hirouchi Y,Li XH,Kitamoto S,Takeshita A,Egashira K

更新日期：2004-08-01 00:00:00

abstract：:Tissue-specific promoters for gene therapy are typically too big for adeno-associated virus (AAV) vectors; thus, the exploration of small effective non-viral regulatory elements is of particular interest. Wild-type AAV can specifically integrate into a region on human chromosome 19 termed AAVS1. Earlier work has deter...

journal_title：Gene therapy

authors： Li C,Hirsch M,Carter P,Asokan A,Zhou X,Wu Z,Samulski RJ

更新日期：2009-01-01 00:00:00

abstract：:Multiple sclerosis (MS) is an immune-mediated disease of the central nervous system (CNS), where suspected autoimmune attack causes nerve demyelination and progressive neurodegeneration and should benefit from both anti-inflammatory and neuroprotective strategies. Although neuroprotection strategies are relatively une...

journal_title：Gene therapy

authors： Baker D,Hankey DJ

更新日期：2003-05-01 00:00:00