A small regulatory element from chromosome 19 enhances liver-specific gene expression.

abstract：:Sinusoidal fenestrae may restrict the transport of gene transfer vectors according to their size. Using Vitrobot technology and cryo-electron microscopy, we show that the diameter of human adenoviral serotype 5 vectors is 93 nm with protruding fibers of 30 nm. Thus, a diameter of fenestrae of 150 nm or more is likely ...

journal_title：Gene therapy

authors： Snoeys J,Lievens J,Wisse E,Jacobs F,Duimel H,Collen D,Frederik P,De Geest B

更新日期：2007-04-01 00:00:00

abstract：:Gene transfer technology has opened novel treatment avenues toward the treatment of damaged musculoskeletal tissues, and may be particularly beneficial to articular cartilage. There is no natural repair mechanism to heal damaged or diseased cartilage. Existing pharmacologic, surgical and cell based treatments may offe...

journal_title：Gene therapy

authors： Trippel SB,Ghivizzani SC,Nixon AJ

更新日期：2004-02-01 00:00:00

abstract：:We explored the possibility of using a genetic approach to inhibit integrin-mediated endothelial cell adhesion and survival. We constructed recombinant adenoviruses (Ads) expressing chimeric proteins consisting of the cytoplasmic and transmembrane domains of integrin beta1 (CH1), beta3 (CH3) or the beta1 transmembrane...

journal_title：Gene therapy

authors： Oguey D,George PW,Rüegg C

更新日期：2000-08-01 00:00:00

abstract：:Despite significant advances in basic research, the treatment of degenerative diseases of the nervous system remains one of the greatest challenges for translational medicine. The childhood onset motor neuron disorder spinal muscular atrophy (SMA) has been viewed as one of the more tractable targets for molecular ther...

journal_title：Gene therapy

authors： Talbot K,Tizzano EF

更新日期：2017-09-01 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：:Gene modification of malignant cells to express immune stimulators (cytokines and immune costimulators) has provided the basis for a novel form of immunotherapy. Using a MPSV-based retroviral vector with hygromycin resistance gene as a selectable marker, we have studied retrovirus-mediated gene transfer of an immune c...

journal_title：Gene therapy

authors： Hirst WJ,Buggins A,Darling D,Gäken J,Farzaneh F,Mufti GJ

更新日期：1997-07-01 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

authors： Pencavel T,Seth R,Hayes A,Melcher A,Pandha H,Vile R,Harrington KJ

更新日期：2010-08-01 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

authors： Hickson I,Fairbairn LJ,Chinnasamy N,Dexter TM,Margison GP,Rafferty JA

更新日期：1996-10-01 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：:Direct gene transfer for the treatment of human diseases requires a vector which can be administered efficiently, safely and repeatedly. Cationic liposomes represent one of the few examples that can meet these requirements. Currently, more than a dozen cationic liposome formulations have been reported. These liposomes...

journal_title：Gene therapy

authors： Gao X,Huang L

更新日期：1995-12-01 00:00:00

abstract：:The sodium iodide symporter (NIS) mediates iodide uptake into thyrocytes and is the molecular basis of thyroid radioiodine therapy. We previously have shown that NIS gene transfer into the F98 rat gliomas facilitated tumor imaging and increased survival by radioiodine. In this study, we show that: (1) the therapeutic ...

journal_title：Gene therapy

authors： Shen DH,Marsee DK,Schaap J,Yang W,Cho JY,Hinkle G,Nagaraja HN,Kloos RT,Barth RF,Jhiang SM

更新日期：2004-01-01 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：: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

authors： Yang YF,Xue SY,Lu ZZ,Xiao FJ,Yin Y,Zhang QW,Wu CT,Wang H,Wang LS

更新日期：2014-08-01 00:00:00

abstract：:In 2012, we reported that 5 out of 11 subjects in a clinical trial (NCT00372320) administering AdhAQP1 to radiation-damaged parotid glands showed increased saliva flow rates and decreased symptoms over the initial 42 days. AdhAQP1 is a first-generation, E1-deleted, replication-defective, serotype 5 adenoviral vector e...

journal_title：Gene therapy

authors： Zheng C,Baum BJ,Liu X,Goldsmith CM,Perez P,Jang SI,Cotrim AP,McCullagh L,Ambudkar IS,Alevizos I

更新日期：2015-09-01 00:00:00

abstract：:The annual rate of kidney graft loss caused by chronic allograft nephropathy (CAN) has not improved over the past decade. Recent reports suggest that acute renal ischemia results in development of CAN. The goal of the present study was to assess the renoprotective potential and safety of hepatocyte growth factor (HGF)...

journal_title：Gene therapy

authors： Isaka Y,Yamada K,Takabatake Y,Mizui M,Miura-Tsujie M,Ichimaru N,Yazawa K,Utsugi R,Okuyama A,Hori M,Imai E,Takahara S

更新日期：2005-05-01 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

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：:Sustainable correction of severe human genetic disorders of self-renewing tissues, such as the blistering skin disease junctional epidermolysis bullosa (JEB), is facilitated by stable genomic integration of therapeutic genes into somatic tissue stem cells. While integrating viral vectors can achieve this, they suffer ...

journal_title：Gene therapy

authors： Ortiz-Urda S,Lin Q,Yant SR,Keene D,Kay MA,Khavari PA

更新日期：2003-07-01 00:00:00

abstract：:Mucopolysaccharidosis type IIIA (MPS-IIIA) or Sanfilippo A syndrome is a lysosomal storage genetic disease that results from the deficiency of the N-sulfoglucosamine sulfohydrolase (SGSH) protein, a sulfamidase required for the degradation of heparan sulfate glycosaminoglycans (GAGs). The accumulation of these macromo...

journal_title：Gene therapy

authors： Quiviger M,Arfi A,Mansard D,Delacotte L,Pastor M,Scherman D,Marie C

更新日期：2014-12-01 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

authors： Kapsa RM,Quigley AF,Vadolas J,Steeper K,Ioannou PA,Byrne E,Kornberg AJ

更新日期：2002-06-01 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

authors： Piller Puicher E,Tomanin R,Salvalaio M,Friso A,Hortelano G,Marin O,Scarpa M

更新日期：2012-04-01 00:00:00

abstract：:Deficiency in acid alpha-D-glucosidase results in Pompe's disease. Modified single-stranded oligonucleotide (ODN) was designed to correct the acid alpha-D-glucosidase gene with a C1935 --> A (Asp --> Glu) point mutation which causes a complete loss of enzymatic activity for glycogen digestion in the lysosome. The ODN ...

journal_title：Gene therapy

authors： Lu IL,Lin CY,Lin SB,Chen ST,Yeh LY,Yang FY,Au LC

更新日期：2003-10-01 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

authors： Chang SC,Chuang HL,Chen YR,Chen JK,Chung HY,Lu YL,Lin HY,Tai CL,Lou J

更新日期：2003-11-01 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

authors： Ferreira JR,Hirsch ML,Zhang L,Park Y,Samulski RJ,Hu WS,Ko CC

更新日期：2013-02-01 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

authors： Sawyer GJ,Zhang X,Fabre JW

更新日期：2010-04-01 00:00:00

abstract：:We have developed and tested a transfection compound based on synthetic peptides. It consists of a 12 amino acid DNA binding peptide (P2) with an alkyl group added to the aminoterminus (P2lip) and a peptide derived from the hemagglutinin protein (HA). The components aggregate spontaneously to particles that proved to ...

journal_title：Gene therapy

authors： Wilke M,Fortunati E,van den Broek M,Hoogeveen AT,Scholte BJ

更新日期：1996-12-01 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

authors： Wang Z,Tang Z,Zheng Y,Yu D,Spear M,Iyer SR,Bishop B,Wu Y

更新日期：2010-09-01 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

authors： Lamfers ML,Lardenoye JH,de Vries MR,Aalders MC,Engelse MA,Grimbergen JM,van Hinsbergh VW,Quax PH

更新日期：2001-04-01 00:00:00

abstract：:Somatic transgenesis can be used to confer endogenous production of proteins with therapeutic properties. One such product, recombinant soluble human CD4 (sCD4), has been shown to be an efficient inhibitor of human immunodeficiency virus 1 (HIV-1) in vitro, but its too short half-life in vivo has impaired long-term cl...

journal_title：Gene therapy

authors： Valere T,Bohl D,Klatzmann D,Danos O,Sonigo P,Heard JM

更新日期：1995-05-01 00:00:00

abstract：:Recombinant Semliki Forest virus (SFV) is an attractive viral vector system owing to its ability to allow high efficiency of viral protein expression. To produce recombinant pseudotyped human immunodeficiency virus type 1 (HIV-1) virions, we designed a chimeric SFV/HIV vector system that contains both the HIV-1 cis- a...

journal_title：Gene therapy

authors： Del Vecchio C,Calistri A,Lombardi G,Celegato M,Biasolo MA,Palù G,Parolin C

更新日期：2009-02-01 00:00:00