Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product.

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：:Glucagon-like peptide 1 (GLP-1) is released from neuroendocrine cells in the intestine in the postprandial state and augments glucose-stimulated insulin secretion from pancreatic beta cells. To develop non-beta cells that exhibit physiologically regulated insulin secretion, we coexpressed the GLP-1 receptor and human ...

journal_title：Gene therapy

authors： Wu L,Nicholson W,Wu CY,Xu M,McGaha A,Shiota M,Powers AC

更新日期：2003-09-01 00:00:00

abstract：:Interferon-alpha (IFN-alpha) or CD80 transduction of tumor cells individually reduces tumorigenicity and enhances antitumor responses. Here, we report that the combination of IFN-alpha and CD80 cancer gene therapy in poorly immunogenic murine tumor models, the colorectal adenocarcinoma cell line MC38, and the methylch...

journal_title：Gene therapy

authors： Hiroishi K,Tüting T,Tahara H,Lotze MT

更新日期：1999-12-01 00:00:00

abstract：:Sindbis virus expression has been used for in vitro investigations of antigen processing, presentation and epitope mapping. The recent development of a replication-deficient recombinant Sindbis virus expression vector has made in vivo expression possible with minimal pathogenic risk. Advantages of Sindbis virus over o...

journal_title：Gene therapy

authors： Altman-Hamamdzic S,Groseclose C,Ma JX,Hamamdzic D,Vrindavanam NS,Middaugh LD,Parratto NP,Sallee FR

更新日期：1997-08-01 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

authors： Park TK,Wu Z,Kjellstrom S,Zeng Y,Bush RA,Sieving PA,Colosi P

更新日期：2009-07-01 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

authors： Hisayasu S,Miyauchi M,Akiyama K,Gotoh T,Satoh S,Shimada T

更新日期：1999-04-01 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

authors： Ledley FD,McNamee LM,Uzdil V,Morgan IW

更新日期：2014-02-01 00:00:00

abstract：:Intravenous delivery of adenoviruses is the optimal route for many gene therapy applications. Once in the blood, coagulation factor X (FX) binds to the adenovirus capsid and protects the virion from natural antibody and classical complement-mediated neutralisation in mice. However, to date, no studies have examined th...

journal_title：Gene therapy

authors： Duffy MR,Doszpoly A,Turner G,Nicklin SA,Baker AH

更新日期：2016-07-01 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

authors： Jean M,Alameh M,Buschmann MD,Merzouki A

更新日期：2011-08-01 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

authors： Agrawal RS,Muangman S,Layne MD,Melo L,Perrella MA,Lee RT,Zhang L,Lopez-Ilasaca M,Dzau VJ

更新日期：2004-06-01 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

authors： Eliopoulos N,Al-Khaldi A,Beauséjour CM,Momparler RL,Momparler LF,Galipeau J

更新日期：2002-04-01 00:00:00

abstract：:The gene encoding E. coli nitroreductase (NTR) was expressed in the luminal cells of the mammary gland of transgenic mice using the ovine beta-lactoglobulin promoter. Treatment of NTR expressing animals with the prodrug CB1954 (5-aziridin-1-yl-2-4-dinitrobenzamide) resulted in a rapid and selective killing of this pop...

journal_title：Gene therapy

authors： Clark AJ,Iwobi M,Cui W,Crompton M,Harold G,Hobbs S,Kamalati T,Knox R,Neil C,Yull F,Gusterson B

更新日期：1997-02-01 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

authors： Ohnishi N,Itoh K,Itoh Y,Baum C,Higashitsuji H,Yamaguchi K,Tsuji T,Okanoue T,Fujita J

更新日期：2002-02-01 00:00:00

abstract：:Hyperammonemia is less severe in arginase 1 deficiency compared with other urea cycle defects. Affected patients manifest hyperargininemia and infrequent episodes of hyperammonemia. Patients typically suffer from neurological impairment with cortical and pyramidal tract deterioration, spasticity, loss of ambulation, s...

journal_title：Gene therapy

authors： Hu C,Tai DS,Park H,Cantero G,Cantero-Nieto G,Chan E,Yudkoff M,Cederbaum SD,Lipshutz GS

更新日期：2015-02-01 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

authors： Martin JN,Wolken N,Brown T,Dauer WT,Ehrlich ME,Gonzalez-Alegre P

更新日期：2011-07-01 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

authors： Levy RJ,Song C,Tallapragada S,DeFelice S,Hinson JT,Vyavahare N,Connolly J,Ryan K,Li Q

更新日期：2001-05-01 00:00:00

abstract：:Galactose(alpha1,3)galactose on the surface of cells of non-primate organs is the major xenoantigen responsible for hyperacute rejection in xenotransplantation. The antigen is synthesised by (alpha1, 3)galactosyl transferase. Humans lack this enzyme and their serum contains high levels of pre-existing natural antibody...

journal_title：Gene therapy

authors： Jäger U,Takeuchi Y,Porter C

更新日期：1999-06-01 00:00:00

abstract：:Human adenovirus (AV) is a favored vector for delivery of therapeutic genes into certain target cells, such as skeletal muscle cells for gene therapy. Here we show that replication-defective (E1 + E3 deleted) human type 5 adenovirus (AV) recombinants containing a reporter gene insert (RSV-luciferase or RSV-Lux) can ve...

journal_title：Gene therapy

authors： Acsadi G,Jani A,Huard J,Blaschuk K,Massie B,Holland P,Lochmüller H,Karpati G

更新日期：1994-09-01 00:00:00

abstract：:Adenoviral vectors deleted of E1 are attractive vehicles for in vivo gene therapy because efficient gene transfer can be achieved. Immune responses to the vector and vector-transduced cells lead to destruction of target cells, inflammation and difficulties with vector readministration. Immune effectors have been ident...

journal_title：Gene therapy

authors： Jooss K,Turka LA,Wilson JM

更新日期：1998-03-01 00:00:00

abstract：:Prenatal gene therapy has been considered for Herlitz junctional epidermolysis bullosa (H-JEB), a lethal genodermatosis caused by the absence of any of the three subunits of laminin-5, resulting from birth in widespread blistering and erosions of skin and mucosae. To investigate this strategy in an animal model, adeno...

journal_title：Gene therapy

authors： Mühle C,Neuner A,Park J,Pacho F,Jiang Q,Waddington SN,Schneider H

更新日期：2006-12-01 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

authors： Ying Y,Müller OJ,Goehringer C,Leuchs B,Trepel M,Katus HA,Kleinschmidt JA

更新日期：2010-08-01 00:00:00

abstract：:Adenovirus-mediated gene therapy of experimental hepatocarcinoma is hindered by low transduction efficacy in vivo. We evaluated the extent of gene expression following various routes of administration of recombinant adenovirus AdCMVlacZ in diethylnitrosamine-induced rat hepatocarcinoma. We first characterized the vasc...

journal_title：Gene therapy

authors： Gérolami R,Cardoso J,Bralet MP,Cuenod CA,Clément O,Tran PL,Bréchot C

更新日期：1998-07-01 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

authors： Iyer S,Xiao E,Alsayegh K,Eroshenko N,Riggs MJ,Bennett JP Jr,Rao RR

更新日期：2012-05-01 00:00:00

abstract：:Adoptive cell therapy with chimeric antigen receptor (CAR)-modified T cells showed remarkable therapeutic efficacy in the treatment of leukaemia/lymphoma. However, the application to a variety of cancer entities is often constricted by the non-availability of a single chain antibody (scFv), which is usually the target...

journal_title：Gene therapy

authors： Faitschuk E,Nagy V,Hombach AA,Abken H

更新日期：2016-10-01 00:00:00

abstract：:Genetic engineering of T lymphocytes for adoptive clinical immunotherapy calls for efficient gene transduction methods. Therefore, a transient retroviral gene transduction system 'STITCH' was developed comprising pSTITCH retroviral vector encoding the transgene, plasmids encoding Moloney murine leukemia virus gag/pol ...

journal_title：Gene therapy

authors： Weijtens ME,Willemsen RA,Hart EH,Bolhuis RL

更新日期：1998-09-01 00:00:00

abstract：:Urothelial bladder cancer is the most common malignancy of the urinary tract. Although most cases are initially diagnosed as non-muscle-invasive, more than 80% of patients will develop recurrent or metastatic tumors. No effective therapy exists currently for late-stage metastatic tumors. By intravesical application, l...

journal_title：Gene therapy

authors： Zhang KX,Matsui Y,Lee C,Osamu O,Skinner L,Wang J,So A,Rennie PS,Jia WW

更新日期：2016-05-01 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

authors： Rutanen J,Puhakka H,Ylä-Herttuala S

更新日期：2002-11-01 00:00:00

abstract：:Hepatic sequestration of systemically administered adenoviral vectors reduces the number of viral particles available for delivery to other tissues. The biological basis of this phenomenon was investigated using a new in vivo technique which permitted imaging in real time. Recombinant adenovirus serotype 5 knob (Ad5K)...

journal_title：Gene therapy

authors： Zinn KR,Douglas JT,Smyth CA,Liu HG,Wu Q,Krasnykh VN,Mountz JD,Curiel DT,Mountz JM

更新日期：1998-06-01 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

authors： Kawamura I,Morishita R,Tsujimoto S,Manda T,Tomoi M,Tomita N,Goto T,Ogihara T,Kaneda Y

更新日期：2001-06-01 00:00:00

abstract：:Adenovirus-mediated gene transfer of interferon gamma (AdIFN) elicits rejection of intracerebral Lewis lung carcinoma. In this system, gene transfer into brain parenchymal cells is both necessary and sufficient to generate the antitumor response. Despite persistent parenchymal inflammation and demyelination, wild-type...

journal_title：Gene therapy

authors： Fathallah-Shaykh HM,Kafrouni AI,Zhao LJ,Diaz-Arrastia R,Garcia JA,Frawley WH,Forman J

更新日期：2000-12-01 00:00:00