解剖学和形态学
麻醉学
听力与言语-语言病理学
行为科学
心脏和心血管系统
细胞和组织工程学
临床神经病学
危重症监护医学
牙科,口腔外科和医学
皮肤病学
急诊医学
内分泌学和新陈代谢
肠胃学和肝脏学
老人病学和老年医学
卫生保健科学和服务
血液学
免疫学
传染病
综合和补充性医学
医学伦理学
医学信息学
医学实验室技术
医学,全科和内科
医学,法律
医学,研究和试验
神经系统科学
护理
营养学和饮食学
产科医学和妇科医学
肿瘤学
眼科学
整形外科学
耳鼻喉科学
病理学
儿科学
周围血管疾病
药理学和药剂学
生理学
基本医疗保健
精神病学
公共、环境和职业卫生
放射学,核医学和医学成像
康复学
生殖生物学
呼吸系统
风湿病学
运动科学
外科学
毒理学
热带医学
泌尿学和肾脏学
病毒学
老年医学
健康政策和服务
心理学,临床
abstract::We have shown that various forms of oligonucleotides, chimeric RNA-DNA oligonucleotide (RDO) and single-stranded oligodeoxynucleotide (ODN), are capable of chromosomal gene alterations in mammalian cells. Using two ODNs we corrected an inactivating mutation in the tyrosinase gene and introduced an activating mutation ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301862
更新日期:2002-12-01 00:00:00
abstract::Duchenne muscular dystrophy (DMD) is an X-linked, lethal muscular disorder caused by a defect in the DMD gene. AAV vector-mediated micro-dystrophin cDNA transfer is an attractive approach to treatment of DMD. To establish effective gene transfer into skeletal muscle, we examined the transduction efficiency of an AAV v...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301829
更新日期:2002-12-01 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
pub_type: 杂志文章,评审
doi:10.1038/sj.gt.3301866
更新日期:2002-11-01 00:00:00
abstract::To investigate to what extent myeloablation, graft size, and ex vivo manipulation influence the engraftment and long-term survival of transduced murine hematopoietic cells, groups of C57BL/6J (CD45.2) mice receiving total body irradiation (TBI) (1-9 Gy) or no irradiation were transplanted with either transduced bone m...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301826
更新日期:2002-11-01 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
pub_type: 杂志文章
doi:10.1038/sj.gt.3301800
更新日期:2002-10-01 00:00:00
abstract::Intramuscular (i.m.) injection of plasmids followed by electropermeabilization is an efficient process to deliver genes into skeletal myofibers that permits proteins to be produced and secreted at therapeutically relevant levels. To further improve skeletal muscle as a bioreactor, we identified a formulation that elev...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301806
更新日期:2002-10-01 00:00:00
abstract::Most neoplasms do not induce antitumor immune responses that can control tumor growth. Tumor associated antigens (TAAs) are insufficiently immunogenic. A vaccine that augments the immunogenic properties of TAAs could be of importance in the treatment of cancer patients. In an animal model, we prepared a vaccine by tra...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301765
更新日期:2002-09-01 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
pub_type: 杂志文章
doi:10.1038/sj.gt.3301786
更新日期:2002-09-01 00:00:00
abstract::Recently, there has been an increasing level of interest in electroporation for gene delivery due to the site-specific nature of the delivery, as well as the high efficiency of the method. Electroporation involves the application of a pulsed electric field to cells to enhance cell permeability, resulting in the transi...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301733
更新日期:2002-08-01 00:00:00
abstract::To identify the intracellular barriers to efficient gene transfer, we studied the intracellular trafficking of biotinylated plasmid DNA complexed with either fluorescein-conjugated lactosylated or mannosylated polylysine by confocal microscopy. Both are known to be taken up by cystic fibrosis airway epithelial cells (...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301768
更新日期:2002-08-01 00:00:00
abstract::We have previously synthesized a new cationic liposome that displays high efficiency and low toxicity, 3 beta[l-ornithinamide-carbamoyl] cholesterol (O-Chol), using solid-phase synthesis. In this study, O-Chol was applied to in vitro and in vivo models of ovarian cancer. Intraperitoneal gene delivery for peritoneal di...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301704
更新日期:2002-07-01 00:00:00
abstract::Gene transfer to airway epithelia with amphotropic pseudotyped retroviral vectors is inefficient following apical vector application. To better understand this inefficiency, we localized the expression of Pit2, the amphotropic receptor, in polarized human airway epithelia. Pit2 was expressed on both the apical and bas...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301714
更新日期:2002-07-01 00:00:00
abstract::We have shown that interleukin-12 (IL-12) generated a strong, albeit transient, anti-tumor response, mostly mediated by natural killer (NK) cell. T cell participation, in addition to NK cells, was essential for persistence of the anti-tumor response. Ligation of 4-1BB, a co-stimulatory receptor expressed on activated ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301687
更新日期:2002-06-01 00:00:00
abstract::The recent surge of DNA sequence information resulting from the efforts of agencies interested in deciphering the human genetic code has facilitated technological developments that have been critical in the identification of genes associated with numerous disease pathologies. In addition, these efforts have opened the...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301740
更新日期:2002-06-01 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
pub_type: 杂志文章
doi:10.1038/sj.gt.3301737
更新日期:2002-06-01 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
pub_type: 杂志文章
doi:10.1038/sj.gt.3301738
更新日期:2002-06-01 00:00:00
abstract::The use of adenoviral vectors as potent gene delivery systems requires expression of the Coxsackievirus/adenovirus receptor (CVADR) on the target cell surface. This receptor is important for virus attachment to the cell surface. For effective internalization of the vector into the target cell the integrins alpha(v)bet...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301689
更新日期:2002-05-01 00:00:00
abstract::The existence of cells with stem cell-like abilities derived from various tissues can now be extended to include the skeletal muscle compartment. Although researchers have focused on the utilization of these cells with regard to their myogenic capacity, initially exploring more efficient cellular therapy treatments fo...
journal_title:Gene therapy
pub_type: 杂志文章,评审
doi:10.1038/sj.gt.3301719
更新日期:2002-05-01 00:00:00
abstract::Naturally occurring drug resistance genes of human origin can be exploited for selection of genetically engineered cells co-expressing a desired therapeutic transgene. Their non-immunogenicity in clinical applications would be a major asset. Human cytidine deaminase (hCD) is a chemoresistance gene that inactivates cyt...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301675
更新日期:2002-04-01 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
pub_type: 杂志文章
doi:10.1038/sj.gt.3301656
更新日期:2002-03-01 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
pub_type: 杂志文章
doi:10.1038/sj.gt.3301655
更新日期:2002-02-01 00:00:00
abstract::Somatic gene transfer to the pulmonary endothelium may be a useful strategy for modifying the phenotype of endothelium and/or vascular smooth muscle in disorders such as primary pulmonary hypertension, ARDS or pulmonary metastatic disease. Adenoviral (Ad) vectors, although highly efficient in liver gene transfer, have...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301636
更新日期:2002-02-01 00:00:00
abstract::We recently showed that the human telomerase reverse transcriptase (hTERT) promoter induces tumor-specific Bax gene expression and selectively kills various human cancer cells both in vitro and in xenograft tumors. However, it remains unclear whether the hTERT promoter can be used to induce transgene expression in syn...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301619
更新日期:2002-01-01 00:00:00
abstract::Chimeric oligonucleotides have been used successfully to correct point and frameshift mutations in several cell types, as well as in animal and plant models. However, their application to primitive human blood cells has been limited. In this investigation, chimeric oligonucleotides designed to direct a site-specific n...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301610
更新日期:2002-01-01 00:00:00
abstract::Hypoxia initiates an adaptive physiological response in all organisms and plays a role in the pathogenesis of several human diseases. The hypoxia/HIF-inducible factor-1 (HIF-1) transcription factor mediates transcriptional responses to hypoxia by binding to a cis-acting hypoxia-responsive element (HRE) present within ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301605
更新日期:2001-12-01 00:00:00
abstract::Advances in genetic analysis and a greater understanding of human immunodeficiency virus (HIV) molecular pathogenesis have identified critical viral targets for gene interference strategies. RNase P molecules have been proposed as a novel approach for gene targeting based upon their potent catalytic activity, as well ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301606
更新日期:2001-12-01 00:00:00
abstract::We have developed a novel immunostimulatory molecule against tumor cells, composed of an anti-FcgammaRIII (CD16) scFv fused to the platelet-derived growth factor receptor (PDGFR) transmembrane region. This fusion molecule was stably expressed on the tumor cell surface and retained the ability of the parental antibody ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301575
更新日期:2001-11-01 00:00:00
abstract::Both replication-incompetent and replication-selective adenoviruses are being developed for the treatment of cancer and other diseases. Concerns have been raised about the safety of intra-vascular adenovirus administration following a patient death on a clinical trial with a replication-defective adenovirus. In additi...
journal_title:Gene therapy
pub_type: 临床试验,杂志文章
doi:10.1038/sj.gt.3301512
更新日期:2001-11-01 00:00:00
abstract::The progression of chronic renal diseases is considered as an irreversible process that eventually leads to end-stage renal failure characterized by extensive tissue fibrosis. At present, chronic renal fibrosis is incurable and the incidence of affected patients is on the rise worldwide. In this study, we demonstrate ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301545
更新日期:2001-10-01 00:00:00
abstract::Polyethylenimine (PEI) has been studied as an efficient nonviral gene transfer vector. Here, we report the biodistribution fates and safety of plasmid DNA intravenously administered in PEI complexes. Using pCMVbeta as a model gene, the biodistribution of plasmid DNA was measured by quantitative polymerase chain reacti...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301516
更新日期:2001-10-01 00:00:00
abstract::Hepatic fibrosis is a common outcome of chronic liver diseases. In schistosomiasis, chronic parasite egg-induced granuloma formation can lead to fibrosis, which is immunologically characterized by the dominant Th2 response. Recently, it has been shown that gene therapy is an attractive approach for the treatment of he...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301524
更新日期:2001-09-01 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
pub_type: 杂志文章
doi:10.1038/sj.gt.3301538
更新日期:2001-09-01 00:00:00
abstract::The mechanism whereby cationic lipids destabilize cell membranes to facilitate the intracellular delivery of macromolecules such as plasmid DNA or antisense oligonucleotides is not well understood. Here, we show that cationic lipids can destabilize lipid bilayers by promoting the formation of nonbilayer lipid structur...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301506
更新日期:2001-08-01 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
pub_type: 杂志文章
doi:10.1038/sj.gt.3301522
更新日期:2001-08-01 00:00:00
abstract::We introduce a lung inflation-fixation protocol to examine the distribution and gene transfer efficiency of fluorescently tagged lipoplexes using fluorescence confocal microscopy within thick lung tissue sections. Using this technique, we tested the hypothesis that factors related to lipoplex distribution were the pre...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301461
更新日期:2001-06-01 00:00:00
abstract::We have developed synthetic double-stranded oligodeoxynucleotides (ODN) as 'decoy' cis elements that block the binding of nuclear factors to promoter regions of targeted genes, resulting in the inhibition of gene transactivation in vivo. In the present study, we employed decoy ODN targeting the transcription factor nu...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301478
更新日期:2001-06-01 00:00:00
abstract::We have used interleukin (IL)-4 and -10-producing HSV-1 gamma(1)34.5 deletion viruses in gene therapy of a BALB/c model of experimental allergic encephalomyelitis (EAE), a T cell-mediated demyelinating disease of the central nervous system. It is known that in EAE of mice the Th2-type cytokines are down-regulated and ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301465
更新日期:2001-05-01 00:00:00
abstract::Gene therapy with viral vectors has progressed to clinical trials. However, the localization of viral vector delivery to diseased target sites remains a challenge. We tested the hypothesis that an adenoviral vector could be successfully delivered by complexation with a specific antibody that is bound to a biodegradabl...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301452
更新日期:2001-05-01 00:00:00
abstract::Injury-induced neointimal development results from migration and proliferation of vascular smooth muscle cells (SMC). Cell migration requires controlled proteolytic degradation of extracellular matrix surrounding the cell. Plasmin is a major contributor to this process by degrading various matrix proteins directly, or...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301437
更新日期:2001-04-01 00:00:00
abstract::Solid tumors meet their demands for nascent blood vessels and increased glycolysis, to combat hypoxia, by activating multiple genes involved in angiogenesis and glucose metabolism. Hypoxia inducible factor-1 (HIF-1) is a constitutively expressed basic helix-loop-helix transcription factor, formed by the assembly of HI...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301388
更新日期:2001-04-01 00:00:00