Efficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders.

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：:Virus-directed enzyme prodrug therapy utilizing the bacterial enzyme nitroreductase delivered by a replication-defective adenovirus vector to activate the prodrug CB1954 is a promising strategy currently undergoing clinical trials in patients with a range of cancers. Similarly, selectively replicating oncolytic adenov...

journal_title：Gene therapy

authors： Palmer DH,Chen MJ,Searle PF,Kerr DJ,Young LS

更新日期：2005-08-01 00:00:00

abstract：:Transplantation of allogeneic pancreatic islets is an effective approach to treat type 1 diabetes. To bypass the need for systemic administration of immunosuppression drugs following transplantation, approaches to genetically modify allogeneic islets to express anti-inflammatory, immunosuppressive, or antiapoptotic pr...

journal_title：Gene therapy

authors： Rehman KK,Wang Z,Bottino R,Balamurugan AN,Trucco M,Li J,Xiao X,Robbins PD

更新日期：2005-09-01 00:00:00

abstract：:RNA interference (RNAi) was reported to block hepatitis B virus (HBV) gene expression and replication in vitro and in vivo. However, it remains a technical challenge for RNAi-based therapy to achieve long-term and complete inhibition effects in chronic HBV infection, which presumably requires more extensive and unifor...

journal_title：Gene therapy

authors： Chen CC,Ko TM,Ma HI,Wu HL,Xiao X,Li J,Chang CM,Wu PY,Chen CH,Han JM,Yu CP,Jeng KS,Hu CP,Tao MH

更新日期：2007-01-01 00:00:00

abstract：:The production of high-titer recombinant adeno-associated virus (rAAV) vector is essential for treatment of genetic diseases affecting the retina and choroid, where anatomical constraints may limit injectable volumes. Problematically, cytotoxicity arising from overexpression of the transgene during vector production f...

journal_title：Gene therapy

authors： Reid CA,Boye SL,Hauswirth WW,Lipinski DM

更新日期：2017-08-01 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：:Defensins are small cationic proteins that harbor broad-spectrum microbicidal activity against bacteria, fungi and viruses. This study examines the effects on pathogens of the epidermis engineered to express human beta-defensin 3 (HBD3) to combat bacterial infections. First, we examined the localization of HBD3 in the...

journal_title：Gene therapy

authors： Sawamura D,Goto M,Shibaki A,Akiyama M,McMillan JR,Abiko Y,Shimizu H

更新日期：2005-05-01 00:00:00

abstract：:Chemically inducible gene switches that regulate expression of endogenous genes have multiple applications for basic gene expression research and gene therapy. Single-chain zinc-finger transcription factors that utilize either estrogen receptor homodimers or retinoid X receptor-alpha/ecdysone receptor heterodimers are...

journal_title：Gene therapy

authors： Magnenat L,Schwimmer LJ,Barbas CF 3rd

更新日期：2008-09-01 00:00:00

abstract：:A novel class of cationic hyperbranched polymers, containing branched oligoethylenimine (OEI 800 Da) as core, diacrylate esters as linkers and oligoamines as surface modification, was synthesized and evaluated regarding their structure-activity relationship as gene carriers. We show that pseudodendritic core character...

journal_title：Gene therapy

authors： Russ V,Elfberg H,Thoma C,Kloeckner J,Ogris M,Wagner E

更新日期：2008-01-01 00:00:00

abstract：:Successful keratinocyte gene therapy requires the development of efficient methods of gene transfer to keratinocytes. Jet injection of a solution containing DNA can be used to transfer genes to several tissues in vivo. In this article, we tried to introduce DNA into rat and human keratinocytes using this method. First...

journal_title：Gene therapy

authors： Sawamura D,Ina S,Itai K,Meng X,Kon A,Tamai K,Hanada K,Hashimoto I

更新日期：1999-10-01 00:00:00

abstract：:Reactive oxygen species (ROS) have been implicated in the pathogenesis of rheumatoid arthritis (RA), while antioxidant enzymes, such as extracellular superoxide dismutase (EC-SOD) and catalase, block radical-induced events. The present study tested if the ex vivo transfer of EC-SOD and catalase genes alone or in combi...

journal_title：Gene therapy

authors： Dai L,Claxson A,Marklund SL,Feakins R,Yousaf N,Chernajovsky Y,Winyard PG

更新日期：2003-04-01 00:00:00

abstract：:Cationic lipid-based delivery systems such as lipoplexes or stabilized plasmid-lipid particles (SPLP) represent a safer alternative to viral systems for gene therapy applications. We studied the impact of cell cycle status on the efficiency of transfection of human ovarian carcinoma tumor cells using two cationic-lipi...

journal_title：Gene therapy

authors： Mortimer I,Tam P,MacLachlan I,Graham RW,Saravolac EG,Joshi PB

更新日期：1999-03-01 00:00:00

abstract：:The potential for gene therapy to be an effective treatment for cystic fibrosis has been hampered by the limited gene transfer efficiency of current vectors. We have shown that recombinant Sendai virus (SeV) is highly efficient in mediating gene transfer to differentiated airway epithelial cells, because of its capaci...

journal_title：Gene therapy

authors： Ferrari S,Griesenbach U,Iida A,Farley R,Wright AM,Zhu J,Munkonge FM,Smith SN,You J,Ban H,Inoue M,Chan M,Singh C,Verdon B,Argent BE,Wainwright B,Jeffery PK,Geddes DM,Porteous DJ,Hyde SC,Gray MA,Hasegawa M,Alton

更新日期：2007-10-01 00:00:00

abstract：:The clinical efficacy and safety as well as the application range of gene therapy will be broadened by developing systems capable of finely modulating the expression of therapeutic genes. Transgene regulation will be crucial for maintaining appropriate levels of a gene product within the therapeutic range, thus preven...

journal_title：Gene therapy

authors： Toniatti C,Bujard H,Cortese R,Ciliberto G

更新日期：2004-04-01 00:00:00

abstract：:Retroviral vectors are being used increasingly in clinical gene therapy protocols but low transduction frequencies are presenting a significant obstacle to progress. In this paper we report a simple method to enhance the efficiency of ex vivo retroviral gene transfer. Calcium chloride is added to the vector stock and ...

journal_title：Gene therapy

authors： Morling FJ,Russell SJ

更新日期：1995-09-01 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

authors： Li HL,Li S,Shao JY,Lin XB,Cao Y,Jiang WQ,Liu RY,Zhao P,Zhu XF,Zeng MS,Guan ZZ,Huang W

更新日期：2008-02-01 00:00:00

abstract：:Gene transfer complexes containing poly-L-lysine (poly-K) and DNA with ligands directed at the serpin enzyme complex receptor (sec-R) deliver reporter genes to receptor-bearing cells in vivo. Expression lasts for about 30 days, when complexes containing long-chain poly-K are used. Extending the duration of expression ...

journal_title：Gene therapy

authors： Ziady AG,Kim J,Colla J,Davis PB

更新日期：2004-09-01 00:00:00

abstract：:Plasmids carrying the Epstein-Barr virus (EBV) latent gene EBNA1 and the EBV latent origin of replication (oriP) stay in transfected human cells as autonomously replicating extrachromosomal genetic units. They thus might represent a suitable tool for cytokine gene introduction into human tumor cells with the prospect ...

journal_title：Gene therapy

authors： Mũcke S,Polack A,Pawlita M,Zehnpfennig D,Massoudi N,Bohlen H,Doerfler W,Bornkamm G,Diehl V,Wolf J

更新日期：1997-02-01 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：: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

authors： Asselin-Paturel C,Lassau N,Guinebretière JM,Zhang J,Gay F,Bex F,Hallez S,Leclere J,Peronneau P,Mami-Chouaib F,Chouaib S

更新日期：1999-04-01 00:00:00

abstract：:Introduction of the herpes simplex virus type 1 thymidine kinase gene into tumor cells, followed by the administration of the antiherpes nucleoside analogue ganciclovir has been demonstrated to be effective in eliminating solid tumors in animals. The success of this combination treatment largely depends on the bystand...

journal_title：Gene therapy

authors： Degrève B,De Clercq E,Balzarini J

更新日期：1999-02-01 00:00:00

abstract：:Highly active antiretroviral therapy (HAART) treatment for HIV has changed the course of AIDS in societies in which the drugs are readily available. Despite the great success of HAART, drug resistance and toxicity issues still remain a concern for some individuals. Thus, a number of investigators have been exploring o...

journal_title：Gene therapy

authors： Zhou J,Rossi JJ

更新日期：2011-12-01 00:00:00

abstract：:Catheter-based percutaneous transluminal gene delivery (PTGD) into the coronary artery still falls behind the expectations of an efficient myocardial gene delivery system. In this study gene delivery was applied by selective pressure-regulated retroinfusion through the coronary veins to prolong adhesion of replication...

journal_title：Gene therapy

authors： Boekstegers P,von Degenfeld G,Giehrl W,Heinrich D,Hullin R,Kupatt C,Steinbeck G,Baretton G,Middeler G,Katus H,Franz WM

更新日期：2000-02-01 00:00:00

abstract：:A simple, efficient and reproducible method to transduce proteins into mammalian cells has not been established. Here we describe a novel protein transduction method based on a lentiviral vector. We have developed a method to package several thousand foreign protein molecules into a lentivirus-like nanoparticle (LENA)...

journal_title：Gene therapy

authors： Aoki T,Miyauchi K,Urano E,Ichikawa R,Komano J

更新日期：2011-09-01 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：:To induce RNA interference (RNAi), either small interfering RNAs (siRNAs) are directly introduced into the cell or short hairpin RNAs (shRNAs) are expressed from a DNA vector. At present, shRNAs are commonly synthesized by RNA polymerase III (Pol III) promoters of the H1 and U6 RNAs. In this study, we designed and cha...

journal_title：Gene therapy

authors： Choy EY,Kok KH,Tsao SW,Jin DY

更新日期：2008-02-01 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：:A retroviral vector constructed from the murine leukemia virus (MLV) can only express transgenes in cells undergoing mitosis, indicating its suitability as a delivery vehicle for cancer gene therapy. However, the transduction efficiency (TE) of retroviruses embedding endogenous envelope proteins in human cancer cells ...

journal_title：Gene therapy

authors： Lee H,Song JJ,Kim E,Yun CO,Choi J,Lee B,Kim J,Chang JW,Kim JH

更新日期：2001-02-01 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：: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