Abstract:
:We have tested the cationic liposome N-(1-(2,3-dioleoyloxy)propyl)-N,N,N-trimethyl-ammoniummethylsul phate, (DOTAP), for gene delivery in vitro and in vivo with a view to clinical use in gene therapy for cystic fibrosis. Delivery of lacZ cDNA-DOTAP complexes via aerosol showed promoter-dependent differences in the pattern and longevity of expression. Repeated administration was well tolerated. The potential for the transfer of foreign genes into reproductive tissue was investigated by intravenous injection of DNA-DOTAP into female mice. Foreign DNA was undetectable in the ovaries by Southern blot analysis at 1 and 7 days after injection. Our results suggest that DOTAP merits testing in cystic fibrosis patients for delivery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to the respiratory tract and that substitution of the cytomegalovirus (CMV) promoter for the simian virus (SV) promoter may improve on the transitory response reported previously.
journal_name
Gene Therjournal_title
Gene therapyauthors
McLachlan G,Davidson DJ,Stevenson BJ,Dickinson P,Davidson-Smith H,Dorin JR,Porteous DJsubject
Has Abstractpub_date
1995-11-01 00:00:00pages
614-22issue
9eissn
0969-7128issn
1476-5462journal_volume
2pub_type
杂志文章相关文献
GENE THERAPY文献大全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::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
pub_type: 杂志文章,评审
doi:10.1038/sj.gt.3302025
更新日期:2003-05-01 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::Cancer therapies that target a single protein or pathway may be limited by their specificity, thus missing key players that control cellular proliferation and contributing to the failure of the treatment. We propose that approaches to cancer therapy that hit multiple targets would limit the chances of escape. To this ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/s41434-019-0096-1
更新日期:2020-02-01 00:00:00
abstract::Silencing of gene expression by small interfering RNAs (siRNAs) is rapidly becoming a powerful tool for genetic analysis of mammalian cells. The use of DNA-based plasmid vectors to achieve transient and stable expression of siRNA has been developed to avoid the problems of double-stranded oligonucleotides transfection...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302878
更新日期:2007-03-01 00:00:00
abstract::Low levels of expression in haemopoietic cells of the DNA repair protein O6-alkylguanine-DNA alkyltransferase (A Tase), is associated with the dose-limiting sensitivity of these cells to the chemotherapeutic chloroethylating and related methylating agents. Thus, the use of agents which deplete ATase such as O6-benzylg...
journal_title:Gene therapy
pub_type: 杂志文章
doi:
更新日期:1996-10-01 00:00:00
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::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
abstract::Intramuscular (i.m.) injection of a plasmid encoding human carcinoembryonic antigen (CEA) elicited immunity against transplanted syngeneic (C57BL/6) CEA-positive Lewis lung carcinoma (CEA/LLC) cells, but tumors still appeared in all mice. In wild-type mice, coinjection of an IL-12 plasmid markedly enhanced anti-CEA hu...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301274
更新日期:2000-09-01 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::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::The GM2-gangliosidoses are neurological diseases causing premature death, thus developing effective treatment protocols is urgent. GM2-gangliosidoses result from deficiency of a lysosomal enzyme β-hexosaminidase (Hex) and subsequent accumulation of GM2 gangliosides. Genetic changes in HEXA, encoding the Hex α subunit,...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/s41434-019-0120-5
更新日期:2020-05-01 00:00:00
abstract:UNLABELLED:The aim of this study was to determine the effect of RNA interference inhibition of mineralocorticoid receptor (MR) on cold-induced hypertension (CIH) and renal damage. Recombinant adeno-associated virus (AAV) carrying short hairpin small interference (si)RNA for MR (AAV.MR-shRNA) was constructed and tested ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302768
更新日期:2006-07-01 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::A novel synthetic polypeptide designed as a DNA binding-molecule for liver-specific, receptor-mediated, gene transfer was used to selectively introduce reporter genes into liver cells in the form of plasmid DNA-ligand complexes. The polypeptide was a D-lysine/D-serine copolymer (Lys/Ser = 33/36 or 53/60) modified with...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3300868
更新日期:1999-04-01 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::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::This study examines the clinical relevance of tissue engineering integrating gene therapy and polymer science to bone regeneration. Bilateral maxillary defects (3 x 1.2 cm(2)) in 20 miniature swine were bridged with a bioresorbable internal splint. Constructs were created using ex vivo adenovirus bone morphogenetic pr...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302106
更新日期:2003-11-01 00:00:00
abstract::To elucidate further the potential of a Semliki Forest virus (SFV) vector in vivo for gene therapy, we constructed a vector, SFV-IL12, to transfer murine IL-12 genes into tumors. A single intratumoral injection of established B16 murine melanoma with SFV-IL12 resulted in a significant inhibition of tumor growth, while...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3300841
更新日期:1999-04-01 00:00:00
abstract::Cell transplantation holds promise as a potential treatment for cardiac dysfunction. Our group has isolated populations of murine skeletal muscle-derived stem cells (MDSCs) that exhibit stem cell-like properties. Here, we investigated the fate of MDSCs after transplantation into the hearts of dystrophin-deficient mdx ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302521
更新日期:2005-08-01 00:00:00
abstract::In this study, self-inactivating (SIN) retroviral vectors based on feline foamy virus (FFV) were constructed and analysed. The FFV SIN vectors were devoid of the core FFV long terminal repeat promoter plus upstream sequences but contained all structural and regulatory genes. This design allowed sensitive detection of ...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302185
更新日期:2004-03-01 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::The role of two receptors, previously proposed to mediate the entry of adenoviruses into human cells, the coxsackie and adenovirus receptor (CAR) and the major histocompatibility complex (MHC) class I heavy chain has been investigated. The expression of MHC class I in many tumours is reduced or absent, therefore if th...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3301006
更新日期:1999-09-01 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::This study was designed to evaluate the utility of positron emission tomography (PET) to quantify the magnitude and spatial distribution of transgene expression after different methods of adenoviral vector delivery (with surfactant- and saline-based vehicles) within rat lungs. In all, 17 animals (eight in the surfacta...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302117
更新日期:2003-12-01 00:00:00
abstract::This work demonstrates that electrical muscle stimulation markedly increases the transfection efficiency of an intramuscular injection of plasmid DNA. In soleus or extensor digitorum longus muscles of adult rats the percentage of transfected fibers increased from about 1 to more than 10. The number of transfected fibe...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3300847
更新日期:1999-04-01 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
pub_type: 杂志文章,评审
doi:10.1038/sj.gt.3302703
更新日期:2006-03-01 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::Angiogenesis plays a pivotal role in tumor growth, tissue invasion and metastasis. Endostatin is an angiogenesis inhibitor and has been shown to reduce tumor growth in animal models. However, therapy with recombinant endostatin protein was hampered by its short half-life and very-low yield of bioactive protein. We per...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3303038
更新日期:2008-02-01 00:00:00
abstract::In high-risk patients, the ideal cardiovascular gene therapy requires a strategy that provides long-term protection of myocardium against episodes of ischemic/reperfusion injury. We report the development of an efficient, long-lasting pre-emptive gene therapy strategy in a rat model of ischemic-reperfusion (I/R) injur...
journal_title:Gene therapy
pub_type: 杂志文章
doi:10.1038/sj.gt.3302250
更新日期:2004-06-01 00:00:00