Understanding the molecular biology of intervertebral disc degeneration and potential gene therapy strategies for regeneration: a review.

abstract：:Recombinant adeno-associated virus type 2 (rAAV) vectors have recently been used to achieve long-term, high level transduction in vivo. Further development of rAAV vectors for clinical use requires significant technological improvements in large-scale vector production. In order to facilitate the production of rAAV ve...

journal_title：Gene therapy

authors： Conway JE,Rhys CM,Zolotukhin I,Zolotukhin S,Muzyczka N,Hayward GS,Byrne BJ

更新日期：1999-06-01 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：:Although systemic administration of neutralizing anti-TNF antibodies has been used successfully in treating rheumatoid arthritis, there is a potential for side effects. We transduced a collagen reactive T-cell hybridoma with tissue-specific homing properties to assess therapeutic effects of local delivery to inflamed ...

journal_title：Gene therapy

authors： Smith R,Tarner IH,Hollenhorst M,Lin C,Levicnik AU,Fathman CG,Nolan GP

更新日期：2003-08-01 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

authors： Maczuga P,Verheij J,van der Loos C,van Logtenstein R,Hooijer G,Martier R,Borel F,Lubelski J,Koornneef A,Blits B,van Deventer S,Petry H,Konstantinova P

更新日期：2014-01-01 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：:Episomally maintained self-replicating systems present attractive alternative vehicles for gene therapy applications. Recent insights into the ability of chromosomal scaffold/matrix attachment regions (S/MARs) to mediate episomal maintenance of genetic elements allowed the development of a small circular episomal vect...

journal_title：Gene therapy

authors： Papapetrou EP,Ziros PG,Micheva ID,Zoumbos NC,Athanassiadou A

更新日期：2006-01-01 00:00:00

abstract：:We have constructed two recombinant adeno-associated virus (AAV) vectors (pJJ-3GC and pJJ-3ASA) which contained either the human glucocerebrosidase (GC) or arylsulfatase A (ASA) cDNA under the control of an SV40 promoter. These plasmids were co-transfected to 293 cells with a helper plasmid containing trans-acting AAV...

journal_title：Gene therapy

authors： Wei JF,Wei FS,Samulski RJ,Barranger JA

更新日期：1994-07-01 00:00:00

abstract：:We have developed a novel immunostimulatory molecule against tumor cells, composed of an anti-FcgammaRIII (CD16) scFv fused to the platelet-derived growth factor receptor (PDGFR) transmembrane region. This fusion molecule was stably expressed on the tumor cell surface and retained the ability of the parental antibody ...

journal_title：Gene therapy

authors： Gruel N,Fridman WH,Teillaud JL

更新日期：2001-11-01 00:00:00

abstract：:Earlier this laboratory constructed a herpes simplex virus 1 recombinant (R5111) that carries a IL13 ligand inserted into glycoprotein D and can enter cells via the IL13Ralpha2 receptor commonly expressed on the surface of malignant glioma cells. In this report, we describe the properties of two recombinant viruses ca...

journal_title：Gene therapy

authors： Kamiyama H,Zhou G,Roizman B

更新日期：2006-04-01 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：:To identify the intracellular barriers to efficient gene transfer, we studied the intracellular trafficking of biotinylated plasmid DNA complexed with either fluorescein-conjugated lactosylated or mannosylated polylysine by confocal microscopy. Both are known to be taken up by cystic fibrosis airway epithelial cells (...

journal_title：Gene therapy

authors： Grosse S,Tremeau-Bravard A,Aron Y,Briand P,Fajac I

更新日期：2002-08-01 00:00:00

abstract：:Duchenne muscular dystrophy (DMD) is an X-linked, lethal muscular disorder caused by a defect in the DMD gene. AAV vector-mediated micro-dystrophin cDNA transfer is an attractive approach to treatment of DMD. To establish effective gene transfer into skeletal muscle, we examined the transduction efficiency of an AAV v...

journal_title：Gene therapy

authors： Yuasa K,Sakamoto M,Miyagoe-Suzuki Y,Tanouchi A,Yamamoto H,Li J,Chamberlain JS,Xiao X,Takeda S

更新日期：2002-12-01 00:00:00

abstract：:The successful development of adenovirus vectors for vaccines and gene therapy will require a better understanding of the host immune response. Using the ELISPOT assay to measure IFN-gamma-secreting CD8(+) cells, we identify immunodominant epitopes of the adenovirus hexon and DNA-binding protein in BALB/c and C57BL/6 ...

journal_title：Gene therapy

authors： McKelvey T,Tang A,Bett AJ,Casimiro DR,Chastain M

更新日期：2004-05-01 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

authors： Zhang L,Jia TH,Chong AC,Bai L,Yu H,Gong W,Wooley PH,Yang SY

更新日期：2010-10-01 00:00:00

abstract：:The role of gap junctional intercellular communication (GJIC) in bystander killing with herpes simplex virus thymidine kinase (HSV-TK) and ganciclovir (GCV) was evaluated in U251 cells expressing a dominant-negative connexin 43 cDNA (DN14), and in HeLa cells, reportedly devoid of connexin protein. These cell lines bot...

journal_title：Gene therapy

authors： Gentry BG,Im M,Boucher PD,Ruch RJ,Shewach DS

更新日期：2005-07-01 00:00:00

abstract：:The broad host cell range and high expression levels of transgenes are features that have made alphaviruses attractive for gene expression studies and gene therapy applications. Particularly, Semliki Forest virus vectors have been applied for large-scale production of recombinant membrane proteins for drug screening p...

journal_title：Gene therapy

authors： Lundstrom K

更新日期：2005-10-01 00:00:00

abstract：:We have developed a retroviral vector coexpressing the multidrug-resistance 1 (MDR1) cDNA for inducing cancer drug resistance and the truncated version of the low-affinity nerve growth factor receptor (DeltaLNGFR) for cell-surface marking of transduced cells. The vector is based on the FMEV backbone which mediates hig...

journal_title：Gene therapy

authors： Hildinger M,Schilz A,Eckert HG,Bohn W,Fehse B,Zander A,Ostertag W,Baum C

更新日期：1999-07-01 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

authors： Sadat MA,Dirscherl S,Sastry L,Dantzer J,Pech N,Griffin S,Hawkins T,Zhao Y,Barese CN,Cross S,Orazi A,An C,Goebel WS,Yoder MC,Li X,Grez M,Cornetta K,Mooney SD,Dinauer MC

更新日期：2009-12-01 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

authors： Chen S,Shimoda M,Wang MY,Ding J,Noguchi H,Matsumoto S,Grayburn PA

更新日期：2010-11-01 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：:The number of clinical trials using gene transfer technology, either active or under discussion, is increasing rapidly. However, little information is available describing the regulatory procedures or safety specifications that must be considered before initiation of such trials in Europe. We describe the procedure us...

journal_title：Gene therapy

authors： Caplen NJ,Gao X,Hayes P,Elaswarapu R,Fisher G,Kinrade E,Chakera A,Schorr J,Hughes B,Dorin JR

更新日期：1994-03-01 00:00:00

abstract：:The CMV promoter drives high transgene expression and is one of the most commonly used promoters for gene transfer. Tissue-specific mammalian promoters provide an alternative, and it would be useful to have a system to directly compare them to viral promoters free from potential confounding vector-related effects. In ...

journal_title：Gene therapy

authors： Kachi S,Esumi N,Zack DJ,Campochiaro PA

更新日期：2006-05-01 00:00:00

abstract：:Adenovirus (Ad) vectors are of utility for many therapeutic applications. Strategies have been developed to alter adenoviral tropism to achieve a cell-specific gene delivery capacity employing fiber modifications allowing genetic incorporation of targeting motifs. In this regard, single chain antibodies (scFv) represe...

journal_title：Gene therapy

authors： Hedley SJ,Auf der Maur A,Hohn S,Escher D,Barberis A,Glasgow JN,Douglas JT,Korokhov N,Curiel DT

更新日期：2006-01-01 00:00:00

abstract：:A number of genetic disorders are manifested in cutaneous epithelium and gene therapy approaches for treatment of such diseases are being considered. A successful gene therapy protocol requires durable and correctly targeted gene expression within the tissue. The continuous renewal and high levels of compartmentalizat...

journal_title：Gene therapy

authors： Ghazizadeh S,Doumeng C,Taichman LB

更新日期：2002-10-01 00:00:00

abstract：:In view of our recent findings that a truncated form of the envelope (Env) glycoprotein of human immunodeficiency virus type 1 (HIV-1) was efficiently incorporated into MoMLV particles, we studied the generation of Moloney murine leukemia virus (MoMLV)/simian immunodeficiency virus (SIV) pseudotypes. Unlike HIV-1, bot...

journal_title：Gene therapy

authors： Indraccolo S,Minuzzo S,Feroli F,Mammano F,Calderazzo F,Chieco-Bianchi L,Amadori A

更新日期：1998-02-01 00:00:00

abstract：:A highly desirable feature for an human immunodeficiency virus type 1 (HIV-1) vaccine is the ability to induce broadly reactive anti-envelope antibodies that can neutralize primary HIV-1 isolates. Two immunizations with an HIV-1 envelope-encoding plasmid together with recombinant granulocyte-macrophage colony-stimulat...

journal_title：Gene therapy

authors： Rollman E,Hinkula J,Arteaga J,Zuber B,Kjerrström A,Liu M,Wahren B,Ljungberg K

更新日期：2004-07-01 00:00:00

abstract：:Recently, there has been an increasing level of interest in electroporation for gene delivery due to the site-specific nature of the delivery, as well as the high efficiency of the method. Electroporation involves the application of a pulsed electric field to cells to enhance cell permeability, resulting in the transi...

journal_title：Gene therapy

authors： Liu F,Huang L

更新日期：2002-08-01 00:00:00

abstract：:Previous studies have demonstrated that antigen-specific tolerance could be induced by lipopolysaccharide (LPS)-stimulated B cells retrovirally transduced with an immunoglobulin-antigen (or epitope-containing peptide) fusion construct. To investigate the mechanism of this gene therapy system, we now adapted this appro...

journal_title：Gene therapy

authors： Song L,Wang J,Wang R,Yu M,Sun Y,Han G,Li Y,Qian J,Scott DW,Kang Y,Soukhareva N,Shen B

更新日期：2004-10-01 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：:Dendritic cells (DC) are among the most potent antigen-presenting cells known and play an important role in the initiation of antigen-specific T-lymphocyte responses. Several recent studies have demonstrated that DC expressing vector-encoded tumor-associated antigens can induce protective and therapeutic immunity in m...

journal_title：Gene therapy

authors： Diao J,Smythe JA,Smyth C,Rowe PB,Alexander IE

更新日期：1999-05-01 00:00:00