Redirecting adenovirus to pulmonary endothelium by cationic liposomes.

abstract：:We have previously shown that recombinant Sendai virus (SeV) vector, derived from murine parainfluenza virus, is one of the most efficient vectors for airway gene transfer. We have also shown that SeV-mediated transfection on second administration, although reduced by 60% when compared with levels achieved after a sin...

journal_title：Gene therapy

authors： Griesenbach U,McLachlan G,Owaki T,Somerton L,Shu T,Baker A,Tennant P,Gordon C,Vrettou C,Baker E,Collie DD,Hasegawa M,Alton EW

更新日期：2011-02-01 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

authors： Shi Y,Falahati R,Zhang J,Flebbe-Rehwaldt L,Gaensler KM

更新日期：2013-10-01 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

authors： Takahashi M,Katagiri T,Furuya H,Hohjoh H

更新日期：2012-07-01 00:00:00

abstract：:For successful cancer gene therapy via intravenous (i.v.) administration, it is essential to optimize the stability of carriers in the systemic circulation and the cellular association after the accumulation of the carrier in tumor tissue. However, a dilemma exists regarding the use of poly(ethylene glycol) (PEG), whi...

journal_title：Gene therapy

authors： Hatakeyama H,Akita H,Kogure K,Oishi M,Nagasaki Y,Kihira Y,Ueno M,Kobayashi H,Kikuchi H,Harashima H

更新日期：2007-01-01 00:00:00

abstract：:Citrullinemia is an autosomal recessive disorder caused by the deficiency of argininosuccinate synthetase (AS). It is characterized by elevated levels of blood citrulline and ammonia, which often results in hyperammonemic coma and early neonatal death in affected children. We have explored the use of adenoviral vector...

journal_title：Gene therapy

authors： Ye X,Whiteman B,Jerebtsova M,Batshaw ML

更新日期：2000-10-01 00:00:00

abstract：:Insulin gene therapy in clinical medicine is currently hampered by the inability to regulate insulin secretion in a physiological manner, the inefficiency with which the gene is delivered, and the short duration of gene expression. To address these issues, we injected the liver of streptozotocin-induced diabetic rats ...

journal_title：Gene therapy

authors： Kim YD,Park KG,Morishita R,Kaneda Y,Kim SY,Song DK,Kim HS,Nam CW,Lee HC,Lee KU,Park JY,Kim BW,Kim JG,Lee IK

更新日期：2006-02-01 00:00:00

abstract：:For many gene therapy applications the effective titre of retroviral vectors is a limiting factor both in vitro and in vivo. Purification and concentration of retrovirus from packaging cell supernatant can overcome this problem. To this end we have investigated a novel procedure which involves complexing retrovirus to...

journal_title：Gene therapy

authors： Darling D,Hughes C,Galea-Lauri J,Gäken J,Trayner ID,Kuiper M,Farzaneh F

更新日期：2000-06-01 00:00:00

abstract：:The oncotropic phenotypes of several viruses correlate with tumor-associated deficiencies within interferon (IFN) signaling pathways. This observation formed the conceptual basis for developing oncolytic viruses deleted for viral proteins that inhibit the host IFN-dependent antiviral response, such as herpes simplex v...

journal_title：Gene therapy

authors： Sobol PT,Hummel JL,Rodrigues RM,Mossman KL

更新日期：2009-09-01 00:00:00

abstract：:VP22, a structural protein from herpes simplex virus type I, exhibits the unique property of intercellular trafficking. This protein is exported from primary expressing cells and subsequently imported into neighbouring cells. This property is conserved when VP22 is genetically fused to a protein, making it a promising...

journal_title：Gene therapy

authors： Zavaglia D,Favrot MC,Eymin B,Tenaud C,Coll JL

更新日期：2003-02-01 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：: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：:Isolated limb perfusion (ILP) is a limb salvage surgical modality used to deliver chemotherapy and biologic agents to locally advanced and recurrent extremity soft tissue sarcoma (STS), and may be readily tailored for delivery of gene therapy. We set out to test the feasibility of delivering AdFLAGp53 (replication inc...

journal_title：Gene therapy

authors： Hannay J,Davis JJ,Yu D,Liu J,Fang B,Pollock RE,Lev D

更新日期：2007-04-01 00:00:00

abstract：:We have used interleukin (IL)-4 and -10-producing HSV-1 gamma(1)34.5 deletion viruses in gene therapy of a BALB/c model of experimental allergic encephalomyelitis (EAE), a T cell-mediated demyelinating disease of the central nervous system. It is known that in EAE of mice the Th2-type cytokines are down-regulated and ...

journal_title：Gene therapy

authors： Broberg E,Setälä N,Röyttä M,Salmi A,Erälinna JP,He B,Roizman B,Hukkanen V

更新日期：2001-05-01 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：:Autonomous parvoviruses are small, single strand DNA viruses which preferentially replicate in transformed and tumor cells, causing cell death by expression of the cytotoxic nonstructural protein, NS1. Several parvoviruses of the rodent group, including LuIII, efficiently infect human transformed cell lines. The poten...

journal_title：Gene therapy

authors： Maxwell IH,Maxwell F

更新日期：1999-03-01 00:00:00

abstract：:We have comparatively evaluated the efficiency of a series of retroviral vectors transducing the gp91-phox gene, whose defects are responsible for impaired production of superoxide anion (O2-) by phagocytic cells and lead to the X-linked form of chronic granulomatous disease (X-CGD). These vectors included four constr...

journal_title：Gene therapy

authors： Zentilin L,Qin G,Tafuro S,Dinauer MC,Baum C,Giacca M

更新日期：2000-01-01 00:00:00

abstract：:Intervertebral disc degeneration (IVDD) is a multi-factorial process characterized by phenotypic and genotypic changes, which leads to low back pain and disability. Prolonged imbalance between anabolism and catabolism in discs alters their composition resulting in progressive loss of proteoglycans and hydration leadin...

journal_title：Gene therapy

authors： Sampara P,Banala RR,Vemuri SK,Av GR,Gpv S

更新日期：2018-04-01 00:00:00

abstract：:Virotherapy is currently being developed for many different types of viruses including replication-competent murine leukaemia virus (MLV) as a novel tool in cancer therapy. However, there is the risk of insertional mutagenesis associated with this virus, making careful preclinical studies necessary before its first ap...

journal_title：Gene therapy

authors： Duerner LJ,Schwantes A,Schneider IC,Cichutek K,Buchholz CJ

更新日期：2008-11-01 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：: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

authors： Beier R,Hermiston T,Mumberg D

更新日期：2013-01-01 00:00:00

abstract：:We report on systemic delivery and long-term biological effects of apolipoprotein E (apoE) obtained by intramuscular (i.m.) plasmid DNA injection. ApoE plays an important role in lipoprotein catabolism and apoE knock-out mice develop severe hypercholesterolemia and diffuse atherosclerosis. We have injected apoE-defici...

journal_title：Gene therapy

authors： Rinaldi M,Catapano AL,Parrella P,Ciafrè SA,Signori E,Seripa D,Uboldi P,Antonini R,Ricci G,Farace MG,Fazio VM

更新日期：2000-11-01 00:00:00

abstract：:Plasma apolipoprotein AI (apoAI) and lecithin-cholesterol acyltransferase (LCAT) play important roles in reverse cholesterol transport, promoting the removal of excess cholesterol from peripheral cells and reducing formation of atherosclerotic lesions. Gene augmentation of either apoAI or LCAT, or both, are thus attra...

journal_title：Gene therapy

authors： Fan L,Drew J,Dunckley MG,Owen JS,Dickson G

更新日期：1998-10-01 00:00:00

abstract：:Adenoviral gene transfer to hematopoietic stem cells (HSCs)/progenitors would provide a new approach to the treatment of hematopoietic diseases and study of the hematopoietic system. We have previously reported that an adenovirus (Ad) vector composed of whole Ad serotype 35 (Ad35), which belongs to subgroup B, shows e...

journal_title：Gene therapy

authors： Sakurai F,Kawabata K,Yamaguchi T,Hayakawa T,Mizuguchi H

更新日期：2005-10-01 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

authors： Zhang L,Zhang T,Wang L,Shao S,Chen Z,Zhang Z

更新日期：2014-07-01 00:00:00

abstract：:Parkinson's disease is a prevalent progressive degenerative disorder of the elderly. There is a current need for novel therapeutic strategies because the standard levodopa pharmacotherapy is only temporarily efficacious. Recently, there have been some high-profile successful preclinical results obtained in animal mode...

journal_title：Gene therapy

authors： Manfredsson FP,Lewin AS,Mandel RJ

更新日期：2006-03-01 00:00:00

abstract：:The development of lentiviral vectors that use Rev-independent mechanisms of nuclear export for their genomic RNA could facilitate the construction of novel anti-HIV vectors. We have improved the titers of Rev-independent lentiviral vectors having the SRV-1 CTE by mutating the major splice donor and acceptor sites pre...

journal_title：Gene therapy

authors： Mautino MR,Keiser N,Morgan RA

更新日期：2000-08-01 00:00:00

abstract：:We have previously synthesized a new cationic liposome that displays high efficiency and low toxicity, 3 beta[l-ornithinamide-carbamoyl] cholesterol (O-Chol), using solid-phase synthesis. In this study, O-Chol was applied to in vitro and in vivo models of ovarian cancer. Intraperitoneal gene delivery for peritoneal di...

journal_title：Gene therapy

authors： Lee MJ,Cho SS,You JR,Lee Y,Kang BD,Choi JS,Park JW,Suh YL,Kim JA,Kim DK,Park JS

更新日期：2002-07-01 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：:Adenovirus vectors have great potential in cancer gene therapy. Targeting of cancer-testis (CT) antigens, which are specifically presented at the surface of tumor cells by human leukocyte antigen (HLA) class I molecules, is an attractive option. In this study, a single-chain T-cell receptor (scTCR) directed against th...

journal_title：Gene therapy

authors： de Vrij J,Uil TG,van den Hengel SK,Cramer SJ,Koppers-Lalic D,Verweij MC,Wiertz EJ,Vellinga J,Willemsen RA,Hoeben RC

更新日期：2008-07-01 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