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::Patients frequently experience a loss of salivary function following irradiation (IR) for the treatment of an oral cavity and oropharyngeal cancer. Herein, we tested if transfer of fibroblast growth factor-2 (FGF2) cDNA could limit salivary dysfunction after fractionated IR (7.5 or 9 Gy for 5 consecutive days to one p...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2014.63
更新日期:2014-10-01 00:00:00
abstract::Advanced prostate cancer (PC) still remains incurable. Novel immunogene therapy shows promise as treatment strategy that can target both localized and metastasized PC. In this study, we have developed a PC-specific oncolytic adenovirus (Ad-PL-PPT-E1A) armed with fusion gene of prostate-specific antigen and CD40 ligand...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2014.46
更新日期:2014-08-01 00:00:00
abstract::A stable packaging cell line (Vero/BC-F) constitutively expressing fusion (F) protein of the human parainfluenza virus type 2 (hPIV2) was established for production of the F-defective and single round-infectious hPIV2 vector in a strategy for recombinant vaccine development. The F gene expression has not evoked cytost...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2014.55
更新日期:2014-08-01 00:00:00
abstract::Short hairpin RNA (shRNA)-mediated gene regulation is a commonly used technique for gene manipulation. An efficient and safe delivery system is indispensable when shRNA is delivered into living organisms for gene therapy. Previous studies have proved that DNA and protein can be delivered into dendritic cells (DCs) by ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2014.50
更新日期:2014-07-01 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
pub_type: 杂志文章
doi:10.1038/gt.2014.12
更新日期:2014-05-01 00:00:00
abstract::This report examines the commercialization of gene therapy in the context of innovation theories that posit a relationship between the maturation of a technology through its life cycle and prospects for successful product development. We show that the field of gene therapy has matured steadily since the 1980s, with th...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2013.72
更新日期:2014-02-01 00:00:00
abstract::Mutations in GJB2, which codes for the gap junction (GJ) protein connexin26 (Cx26), are the most common causes of human nonsyndromic hereditary deafness. We inoculated modified adeno-associated viral (AAV) vectors into the scala media of early postnatal conditional Gjb2 knockout mice to drive exogenous Cx26 expression...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2013.59
更新日期:2014-01-01 00:00:00
abstract::Constitutive expression of short hairpin RNAs (shRNAs) may cause cellular toxicity in vivo and using microRNA (miRNA) scaffolds can circumvent this problem. Previously, we have shown that embedding small interfering RNA sequences targeting apolipoprotein B100 (ApoB) in shRNA (shApoB) or miRNA (miApoB) scaffolds result...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2013.58
更新日期:2014-01-01 00:00:00
abstract::Urea cycle defects presenting in the neonatal period with hyperammonaemia are associated with high morbidity and mortality, and necessitate liver transplantation for long-term management. Gene therapy is therefore an attractive possibility, with vectors based on adeno-associated virus (rAAV) currently showing exciting...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2013.51
更新日期:2013-12-01 00:00:00
abstract::Citrullinemia type 1 (CTLN1) is an autosomal recessive disorder of metabolism caused by a deficiency of argininosuccinate synthetase. Despite optimal management, CTLN1 patients still suffer from lethal metabolic instability and experience life-threatening episodes of acute hyperammonemia. A murine model of CTLN1 (fold...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2013.53
更新日期:2013-12-01 00:00:00
abstract::Neonatal AAV8-mediated Factor IX (F.IX) gene delivery was applied as a model for exploring mechanisms of tolerance induction during immune ontogeny. Intraperitoneal delivery of AAV8/ Factor IX (hF.IX) during weeks 1-4 of life, over a 20-fold dose range, directed stable hF.IX expression, correction of coagulopathy in F...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2013.22
更新日期:2013-10-01 00:00:00
abstract::Genetic engineering of T cells for adoptive immunotherapy in cancer patients has shown significant promise. To ensure optimal antitumor activity and safety, the simultaneous expression of multiple genes is frequently required, and short viral-derived 2A sequences are increasingly preferred for this purpose. Concerns e...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2013.25
更新日期:2013-09-01 00:00:00
abstract::Nonhealing bone defects are difficult to treat. As the bone morphogenic protein and transforming growth factor beta pathways have been implicated in bone healing, we hypothesized that percutaneous Smad7 silencing would enhance signaling through both pathways and improve bone formation. Critical sized parietal trephine...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2013.15
更新日期:2013-09-01 00:00:00
abstract::Epstein-Barr virus (EBV)-associated B-cell lymphoproliferative disease (LPD) after hematopoietic stem cell or solid organ transplantation remains a life-threatening complication. Expression of the virus-encoded gene product, EBER, has been shown to prevent apoptosis via blockade of PKR activation. As PKR is a major ce...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2012.93
更新日期:2013-07-01 00:00:00
abstract::Relaxin-3 is a neuropeptide that is abundantly expressed by discrete brainstem neuron populations that broadly innervate forebrain areas rich in the relaxin-3 G-protein-coupled-receptor, RXFP3. Acute and subchronic central administration of synthetic relaxin-3 or an RXFP3-selective agonist peptide, R3/I5, increase fee...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2012.83
更新日期:2013-07-01 00:00:00
abstract::With the long-term goal of developing a gene-based treatment for osteoarthritis (OA), we performed studies to evaluate the equine joint as a model for adeno-associated virus (AAV)-mediated gene transfer to large, weight-bearing human joints. A self-complementary AAV2 vector containing the coding regions for human inte...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2012.81
更新日期:2013-06-01 00:00:00
abstract::Replication-deficient retroviruses have been successfully utilized as vectors, offering an efficient, stable method of therapeutic gene delivery. Many examples exist proving this mode of integrative gene transfer is both effective and safe in cultured systems and clinical trials. Along with their success, severe side ...
journal_title:Gene therapy
pub_type: 杂志文章,评审
doi:10.1038/gt.2012.88
更新日期:2013-06-01 00:00:00
abstract::Oncolytic viruses hold much promise as novel therapeutic agents that can be combined with conventional therapeutic modalities. Measles virus (MV) is known to enter cells using the signaling lymphocyte activation molecule (SLAM), which is expressed on cells of the immune system. Although human breast cancer cell lines ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2012.44
更新日期:2013-03-01 00:00:00
abstract::Conditionally replicating adenoviruses (CRAd) are a promising class of gene therapy agents that can overcome already known glioblastoma (GBM) resistance mechanisms but have limited distribution upon direct intratumoral (i.t.) injection. Collagen bundles in the extracellular matrix (ECM) have an important role in inhib...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2012.42
更新日期:2013-03-01 00:00:00
abstract::Multipotent adult progenitor cells (MAPCs) are bone marrow-derived stem cells with a high growth rate suitable for therapeutical applications as three-dimensional (3D) aggregates. Combined applications of osteogenically differentiated MAPC (OD-MAPC) aggregates and adeno-associated viral vectors (AAV) in bone bioengine...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2012.16
更新日期:2013-02-01 00:00:00
abstract::Newcastle disease virus (NDV) is an oncolytic paramyxovirus with a nonsegmented single-stranded RNA genome. In this report, a recombinant oncolytic NDV was passaged in human tumor xenografts and reisolated and characterized after two rounds of bioselection. Several isolates could be recovered that differed from the pa...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2012.13
更新日期:2013-01-01 00:00:00
abstract::Signal transducer and activator of transcription 3 (STAT3) is constitutively activated in diverse cancers, which contributes to the proliferation and survival of cancer cells by upregulating apoptosis inhibitors and cell cycle regulators. Suppressor of cytokine signaling 1 (SOCS1) is an important negative regulator of...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2012.4
更新日期:2013-01-01 00:00:00
abstract::The efficient delivery of genetic material to the developing fetal brain represents a powerful research tool and a means to supply therapy in a number of neonatal lethal neurological disorders. In this study, we have delivered vectors based upon adenovirus serotype 5 (Ad5) and adeno-associated virus (AAV) pseudotypes ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.157
更新日期:2012-09-01 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
pub_type: 杂志文章
doi:10.1038/gt.2011.193
更新日期:2012-07-01 00:00:00
abstract::Varying degrees of metabolic abnormalities mediated by chronic inflammation are implicated in the chronic glomerular injuries associated with obesity. Interleukin (IL)-10, a pleiotropic cytokine, exerts anti-inflammatory effects in numerous biological settings. In the present study, we explored the biological benefits...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.183
更新日期:2012-05-01 00:00:00
abstract::Human pluripotent stem cell-derived neural progenitor (hNP) cells are an excellent resource for understanding early neural development and neurodegenerative disorders. Given that many neurodegenerative disorders can be correlated with defects in the mitochondrial genome, optimal utilization of hNP cells requires an ab...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.134
更新日期:2012-05-01 00:00:00
abstract::Mucopolysaccharidosis type I (MPSI) is an autosomic recessive, lysosomal storage disorder due to the deficit of the enzyme α-L-iduronidase (IDUA). The disease accounts for a general impairment of tissue and organ functions, mainly including heart disease, corneal clouding, organomegaly, skeletal malformations and join...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.94
更新日期:2012-04-01 00:00:00
abstract::Partial resistance of primary mouse hepatocytes to lentiviral (LV) vector transduction poses a challenge for ex vivo gene therapy protocols in models of monogenetic liver disease. We thus sought to optimize ex vivo LV gene transfer while preserving the hepatocyte integrity for subsequent transplantation into recipient...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.117
更新日期:2012-04-01 00:00:00
abstract::Substantial advances in our understanding of lentivirus lifecycles and their various constituent proteins have permitted the bioengineering of lentiviral vectors now considered safe enough for clinical trials for both lethal and non-lethal diseases. They possess distinct properties that make them particularly suitable...
journal_title:Gene therapy
pub_type: 杂志文章,评审
doi:10.1038/gt.2011.153
更新日期:2012-02-01 00:00:00
abstract::Cell surface targeting of recombinant adeno-associated virus (rAAV) vectors is an attractive strategy to modify AAV's natural tropism. As modification of the capsid surface is likely to affect the mechanism of vector internalization and consequently the vector's intracellular fate, we investigated early steps in cell ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.78
更新日期:2012-02-01 00:00:00
abstract::Coagulation factor X (FX)-binding ablated adenovirus type 5 (Ad5) vectors have been genetically engineered to ablate the interaction with FX, resulting in substantially reduced hepatocyte transduction following intravenous administration in rodents. Here, we quantify viral genomes and gene transfer mediated by Ad5 and...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.87
更新日期:2012-01-01 00:00:00
abstract::The corticospinal tract (CST) is extensively used as a model system for assessing potential therapies to enhance neuronal regeneration and functional recovery following spinal cord injury (SCI). However, efficient transduction of the CST is challenging and remains to be optimised. Recombinant adeno-associated viral (A...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.71
更新日期:2012-01-01 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
pub_type: 杂志文章,评审
doi:10.1038/gt.2011.149
更新日期:2011-12-01 00:00:00
abstract::RNAi represents a powerful technology to specifically downregulate the expression of target genes. For cancer research and therapy, an efficient in vivo delivery system is supposed to distribute RNAi to all tumour cells upon systemic administration. We present replication-competent murine leukaemia virus (MLV) vectors...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.48
更新日期:2011-10-01 00:00:00
abstract::In vitro culture systems of human myogenic cells contribute greatly to elucidation of the molecular mechanisms underlying terminal myogenic differentiation and symptoms of neuromuscular diseases. However, human myogenic cells have limited ability to proliferate in culture. We have established an improved immortalizati...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.44
更新日期:2011-09-01 00:00:00
abstract::A simple, efficient and reproducible method to transduce proteins into mammalian cells has not been established. Here we describe a novel protein transduction method based on a lentiviral vector. We have developed a method to package several thousand foreign protein molecules into a lentivirus-like nanoparticle (LENA)...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.38
更新日期:2011-09-01 00:00:00
abstract::Glucagon-like peptide-1 (GLP-1) is an incretin hormone that regulates blood glucose level post-prandially. It has been proposed that GLP-1 can be used in type 2 diabetes (T2D) mellitus treatment because of its insulinotropic action. Despite its remarkable advantages, GLP-1 suffers the disadvantage of an extremely shor...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.25
更新日期:2011-08-01 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
pub_type: 杂志文章
doi:10.1038/gt.2011.10
更新日期:2011-07-01 00:00:00
abstract::Small interfering RNAs (siRNAs) are emerging as promising therapeutic tools. However, the widespread clinical application of such molecules as modulators of gene expression is still dependent on several aspects that limit their bioavailability. One of the most promising strategies to overcome the barriers faced by gen...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2011.27
更新日期:2011-07-01 00:00:00
abstract::Lentivirus can be engineered to be a highly potent vector for gene therapy applications. However, generation of clinical grade vectors in enough quantities for therapeutic use is still troublesome and limits the preclinical and clinical experiments. As a first step to solve this unmet need we recently introduced a bac...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/gt.2010.162
更新日期:2011-06-01 00:00:00