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::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
pub_type: 杂志文章
doi:10.1038/gt.2011.7
更新日期:2011-06-01 00:00:00
abstract::Because periodontal ligament (PDL) cells are reported to contain progenitor or stem cell populations, they are considered a beneficial cell source for clinical periodontal regeneration. Both bone morphogenetic protein 4 (BMP4) and human telomerase reverse transcriptase (hTERT) have essential roles in the modulation of...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.158
更新日期:2011-05-01 00:00:00
abstract::The umbilical cord provides a rich source of primitive mesenchymal stem cells (human umbilical cord mesenchymal stem cells (HUMSCs)), which have the potential for transplantation-based treatments of Parkinson's Disease (PD). Our pervious study indicated that adenovirus-associated virus-mediated intrastriatal delivery ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.152
更新日期:2011-04-01 00:00:00
abstract::Alveolar bone loss associated with periodontal diseases is the result of osteoclastogenesis induced by bacterial pathogens. The mitogen-activated protein kinase (MAPK) phosphatase 1 (MKP-1) is a critical negative regulator of immune response as a key phosphatase capable of dephosphorylating activated MAPKs. In this st...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.139
更新日期:2011-04-01 00:00:00
abstract::In this study, we describe a simple system in which human keratinocytes can be redirected to an alternative differentiation pathway. We transiently transfected freshly isolated human skin keratinocytes with the single transcription factor OCT4. Within 2 days these cells displayed expression of endogenous embryonic gen...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.148
更新日期:2011-03-01 00:00:00
abstract::Gene targeting by single-stranded oligodeoxyribonucleotides (ssODNs) is a promising technique for introducing site-specific sequence alterations without affecting the genomic organization of the target locus. Here, we discuss the significant progress that has been made over the last 5 years in unraveling the mechanism...
journal_title:Gene therapy
pub_type: 杂志文章,评审
doi:10.1038/gt.2010.161
更新日期:2011-03-01 00:00:00
abstract::We have previously shown that recombinant Sendai virus (SeV) vector, derived from murine parainfluenza virus, is one of the most efficient vectors for airway gene transfer. We have also shown that SeV-mediated transfection on second administration, although reduced by 60% when compared with levels achieved after a sin...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.131
更新日期:2011-02-01 00:00:00
abstract::Adoptive immunotherapy of cancer using chimeric antigen receptor (CAR)-engineered T cells with redirected specificity showed efficacy in recent trials. In preclinical models, 'second-generation' CARs with CD28 costimulatory domain in addition to CD3ζ performed superior in redirecting T-cell effector functions and surv...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.127
更新日期:2011-01-01 00:00:00
abstract::Newly isolated serotypes of AAV readily cross the endothelial barrier to provide efficient transgene delivery throughout the body. However, tissue-specific expression is preferred in most experimental studies and gene therapy protocols. Previous efforts to restrict gene expression to the myocardium often relied on dir...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.105
更新日期:2011-01-01 00:00:00
abstract::Glioblastoma multiforme (GBM) is one of the most formidable brain tumors with a mean survival period of approximately 12 months. To date, a combination of radiotherapy and chemotherapy with an oral alkylating agent, temozolomide (TMZ), has been used as first-line therapy for glioma. However, the efficacy of chemothera...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.88
更新日期:2010-11-01 00:00:00
abstract::This study uses a novel approach to gene therapy in which plasmid DNA is targeted to the pancreas in vivo using ultrasound-targeted microbubble destruction (UTMD) to achieve islet regeneration. Intravenous microbubbles carrying plasmids are destroyed within the pancreatic microcirculation by ultrasound, achieving loca...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.85
更新日期:2010-11-01 00:00:00
abstract::Both atherosclerosis and arterial interventions induce oxidative stress mediated in part by nicotinamide adenine dinucleotide phosphate (NADPH) oxidases that have a pivotal role in the development of neointimal hyperplasia and restenosis. For small interfering RNA (siRNA) targeting of the NOX2 (Cybb) component of the ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.69
更新日期:2010-10-01 00:00:00
abstract::Chimeric antigen receptors (CARs, immunoreceptors) are frequently used to redirect T cells with pre-defined specificity, in particular towards tumour cells for use in adoptive immunotherapy of malignant diseases. Specific targeting is mediated by an extracellularly located antibody-derived binding domain, which is joi...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.91
更新日期:2010-10-01 00:00:00
abstract::Exogenous osteoprotegerin (OPG) gene modification appears a therapeutic strategy for osteolytic aseptic loosening. The feasibility and efficacy of a cell-based OPG gene delivery approach were investigated using a murine model of knee prosthesis failure. A titanium pin was implanted into mouse proximal tibia to mimic a...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.64
更新日期:2010-10-01 00:00:00
abstract::Persistence of human immunodeficiency virus (HIV) despite highly active antiretroviral therapy (HAART) is a lasting challenge to virus eradication. To develop a strategy complementary to HAART, we constructed a series of Rev-dependent lentiviral vectors carrying diphtheria toxin A chain (DT-A) and its attenuated mutan...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.53
更新日期:2010-09-01 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
pub_type: 杂志文章
doi:10.1038/gt.2010.44
更新日期:2010-08-01 00:00:00
abstract::Viral therapy of cancer includes strategies such as viral transduction of tumour cells with 'suicide genes', using viral infection to trigger immune-mediated tumour cell death and using oncolytic viruses for their direct anti-tumour action. However, problems still remain in terms of adequate viral delivery to tumours....
journal_title:Gene therapy
pub_type: 杂志文章,评审
doi:10.1038/gt.2010.48
更新日期:2010-08-01 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
pub_type: 杂志文章
doi:10.1038/gt.2010.49
更新日期:2010-08-01 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
pub_type: 杂志文章
doi:10.1038/gt.2010.40
更新日期:2010-07-01 00:00:00
abstract::To test whether fast-acting, self-complimentary (sc), adeno-associated virus-mediated RPE65 expression prevents cone degeneration and/or restores cone function, we studied two mouse lines: the Rpe65-deficient rd12 mouse and the Rpe65-deficient, rhodopsin null ('that is, cone function-only') Rpe65(-/-)::Rho(-/-) mouse....
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.29
更新日期:2010-07-01 00:00:00
abstract::Bone marrow presents an attractive option for the treatment of articular cartilage defects as it is readily accessible, it contains mesenchymal progenitor cells that can undergo chondrogenic differentiation and, once coagulated, it provides a natural scaffold that contains the cells within the defect. This study was p...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.16
更新日期:2010-06-01 00:00:00
abstract::Advances in islet transplantation have encouraged efforts to create alternative insulin-secreting cells that overcome limitations associated with current therapies. We have recently demonstrated durable correction of murine and porcine diabetes by syngeneic and autologous implantation, respectively, of primary hepatoc...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.12
更新日期:2010-05-01 00:00:00
abstract::Hydrodynamic gene delivery to the liver is an attractive approach for clinical liver gene therapy, but critical aspects of technique remain uncertain. There has not been to date any report of high levels of hydrodynamic gene delivery to the liver, except in rodents. Regional hydrodynamic delivery to individual lobes/s...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.167
更新日期:2010-04-01 00:00:00
abstract::Gene therapy is an attractive method for the treatment of cardiovascular disease. However, using current strategies, induction of gene expression at therapeutic levels is often inefficient. In this study, we show a novel electroporation (EP) method to enhance the delivery of a plasmid expressing an angiogenic growth f...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.153
更新日期:2010-03-01 00:00:00
abstract::Glucagon-like peptide-1 (GLP-1) is an incretin hormone that performs a wide array of well-characterized antidiabetic actions, including stimulation of glucose-dependent insulin secretion, upregulation of insulin gene expression and improvements in beta-cell survival. GLP-1-receptor agonists have been developed for tre...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.143
更新日期:2010-02-01 00:00:00
abstract::The pressing challenge for contemporary gene therapy is to deliver enough therapeutic genes to enough cancer cells in vivo. With the aim of improving viral distribution and tumor penetration, we explored the use of decorin to enhance viral spreading and tumor tissue penetration. We generated decorin-expressing replica...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.142
更新日期:2010-02-01 00:00:00
abstract::A fully intact immune system would be expected to hinder the efficacy of oncolytic virotherapy by inhibiting viral replication. Simultaneously, however, it may also enhance antitumor therapy through initiation of proinflammatory, antiviral cytokine responses at the tumor site. The aim of this study was to investigate ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.161
更新日期:2010-02-01 00:00:00
abstract::Platelet-derived growth factor-BB (PDGF-BB) stimulates repair of healing-impaired chronic wounds such as diabetic ulcers and periodontal lesions. However, limitations in predictability of tissue regeneration occur due, in part, to transient growth factor bioavailability in vivo. Here, we report that gene delivery of P...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.117
更新日期:2010-01-01 00:00:00
abstract::X-linked chronic granulomatous disease (X-CGD) is an inherited immunodeficiency with absent phagocyte NADPH-oxidase activity caused by defects in the gene-encoding gp91(phox). Here, we evaluated strategies for less intensive conditioning for gene therapy of genetic blood disorders without selective advantage for gene ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.96
更新日期:2009-12-01 00:00:00
abstract::Adenovirus (Ad)-mediated delivery of anti-angiogenic molecules into tumors constitutes an appealing approach for growth inhibition. However, lack of expression on tumors of Ad receptors leads to weak tumor transduction. Therefore, to provide Ad with a new entry pathway into tumors, an NGR peptide was inserted into eit...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.97
更新日期:2009-12-01 00:00:00
abstract::For certain gene therapy applications, the simultaneous delivery of multiple genes would allow for novel therapies. In the case of adeno-associated virus (AAV) vectors, the limited packaging capacity greatly restricts current methods of carrying multiple transgene cassettes. To address this issue, a recombinant AAV (r...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.106
更新日期:2009-11-01 00:00:00
abstract::In the last two decades, remarkable advances have been made in the development of technologies used to engineer new aptamers and ribozymes. This has encouraged interest among researchers who seek to create new types of gene-control systems that can be made to respond specifically to small-molecule signals. Validation ...
journal_title:Gene therapy
pub_type: 杂志文章,评审
doi:10.1038/gt.2009.81
更新日期:2009-10-01 00:00:00
abstract::The oncotropic phenotypes of several viruses correlate with tumor-associated deficiencies within interferon (IFN) signaling pathways. This observation formed the conceptual basis for developing oncolytic viruses deleted for viral proteins that inhibit the host IFN-dependent antiviral response, such as herpes simplex v...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.68
更新日期:2009-09-01 00:00:00
abstract::Growth factor therapy is an emerging treatment modality that enhances tissue vascularization, promotes healing and regeneration and can treat a variety of inflammatory diseases. Both recombinant human growth factor proteins and their gene therapy are in human clinical trials to heal chronic wounds. As platelet-derived...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.60
更新日期:2009-09-01 00:00:00
abstract::X-linked juvenile retinoschisis (XLRS) is a neurodevelopmental abnormality caused by retinoschisin gene mutations. XLRS is characterized by splitting through the retinal layers and impaired synaptic transmission of visual signals resulting in impaired acuity and a propensity to retinal detachment. Several groups have ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.61
更新日期:2009-07-01 00:00:00
abstract::Choroidal neovascularization (CNV) leads to loss of vision in age-related macular degeneration (AMD), the leading cause of blindness in adult population over 50 years old. In this study, we developed intravenously administered, nanoparticulate, targeted nonviral retinal gene delivery systems for the management of CNV....
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2008.185
更新日期:2009-05-01 00:00:00
abstract::Pancreatic cancer and chronic pancreatitis are clinical syndromes associated with severe pain that is difficult to manage. Thus, seeking additional pain reduction therapies is warranted. Excessive alcohol consumption over an extended period of time is the primary causal agent in pancreatitis. The efficacy of a replica...
journal_title:Gene therapy
pub_type: 杂志文章,评审
doi:10.1038/gt.2009.27
更新日期:2009-04-01 00:00:00
abstract::First-generation adenoviral (Ad) vectors are frequently used vectors for experimental and clinical gene transfer. Earlier it has been shown that parallel overexpression of the cell cycle regulator p21(Waf1/Cip1) (p21) or antiapoptotic bcl-2 from a second vector reduces cytotoxicity and improves transgene expression. H...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2009.2
更新日期:2009-04-01 00:00:00
abstract::Human adipose tissue mesenchymal stromal cells (AMSCs) share common traits, including similar differentiation potential and cell surface markers, with their bone marrow counterparts. Owing to their general availability, higher abundance and ease of isolation AMSCs may be convenient autologous delivery vehicles for loc...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2008.176
更新日期:2009-04-01 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
pub_type: 杂志文章
doi:10.1038/gt.2008.181
更新日期:2009-03-01 00:00:00