Adenovirus-mediated expresssion of the murine ecotropic receptor facilitates transduction of human hematopoietic cells with an ecotropic retroviral vector.

abstract：:Synthetic oligonucleotides and DNA fragments of less than 1 kilobase (kb) have been shown to cause site-specific genetic alterations in mammalian cells in culture and in vivo. We have used a lacZ reporter gene system to compare the efficiency of episomal and chromosomal gene repair in human embryonic kidney epithelial...

journal_title：Gene therapy

authors： Nickerson HD,Colledge WH

更新日期：2003-09-01 00:00:00

abstract：:Small interfering RNAs (siRNAs) are emerging as promising therapeutic tools. However, the widespread clinical application of such molecules as modulators of gene expression is still dependent on several aspects that limit their bioavailability. One of the most promising strategies to overcome the barriers faced by gen...

journal_title：Gene therapy

authors： Dharmapuri S,Peruzzi D,Marra E,Palombo F,Bett AJ,Bartz SR,Yong M,Ciliberto G,La Monica N,Buser CA,Toniatti C,Aurisicchio L

更新日期：2011-07-01 00:00:00

abstract：:A major concern of using viral gene therapy is the potential for uncontrolled vector propagation and infection that might result in serious deleterious effects. To enhance the safety, several viral vectors, including vectors based on Sindbis virus, were engineered to lose their capability to replicate and spread after...

journal_title：Gene therapy

authors： Tseng JC,Daniels G,Meruelo D

更新日期：2009-02-01 00:00:00

abstract：:Aptamers are oligonucleotides evolved in vitro or in nature to bind target ligands with high affinity and specificity. They are emerging as powerful tools in the fields of therapeutics, drug development, target validation and diagnostics. Aptamers are attractive alternatives to antibody- and small-molecule-based thera...

journal_title：Gene therapy

authors： Que-Gewirth NS,Sullenger BA

更新日期：2007-02-01 00:00:00

abstract：:The progression of chronic renal diseases is considered as an irreversible process that eventually leads to end-stage renal failure characterized by extensive tissue fibrosis. At present, chronic renal fibrosis is incurable and the incidence of affected patients is on the rise worldwide. In this study, we demonstrate ...

journal_title：Gene therapy

authors： Yang J,Dai C,Liu Y

更新日期：2001-10-01 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：:Cancer cachexia, characterized by anorexia, weight loss and progressive tissue wasting, has been postulated to be mediated by various cytokines. However, the precise mechanism of cachexia induction is not fully explained. We have developed synthetic double-stranded oligodeoxynucleotides (ODN) as 'decoy' cis-elements t...

journal_title：Gene therapy

authors： Kawamura I,Morishita R,Tomita N,Lacey E,Aketa M,Tsujimoto S,Manda T,Tomoi M,Kida I,Higaki J,Kaneda Y,Shimomura K,Ogihara T

更新日期：1999-01-01 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：:Genetic engineering of T cells for adoptive immunotherapy in cancer patients has shown significant promise. To ensure optimal antitumor activity and safety, the simultaneous expression of multiple genes is frequently required, and short viral-derived 2A sequences are increasingly preferred for this purpose. Concerns e...

journal_title：Gene therapy

authors： Arber C,Abhyankar H,Heslop HE,Brenner MK,Liu H,Dotti G,Savoldo B

更新日期：2013-09-01 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：:During the past decade, both in vitro and in vivo studies have provided new insights into the cellular and molecular mechanisms that govern angiogenesis and arteriogenesis. However, therapeutic angiogenesis clinical trials using recombinant protein or gene therapy formulations of single angiogenic growth factors have ...

journal_title：Gene therapy

authors： Vincent KA,Jiang C,Boltje I,Kelly RA

更新日期：2007-05-01 00:00:00

abstract：:Abnormal excitation-contraction coupling is a key pathophysiologic component of heart failure (HF), and at a molecular level reduced expression of the sarcoplasmic reticulum (SR) Ca(2+) ATPase (SERCA2a) is a major contributor. Previous studies in small animals have suggested that restoration of SERCA function is benef...

journal_title：Gene therapy

authors： Byrne MJ,Power JM,Preovolos A,Mariani JA,Hajjar RJ,Kaye DM

更新日期：2008-12-01 00:00:00

abstract：:Rescue of dystrophic skeletal muscle in mdx and utrophin/dystrophin-deficient (dko) mouse models by reintroduction of dystrophin has validated gene therapy as a potential therapeutic approach for Duchenne muscular dystrophy. However, the size of the dystrophin gene exceeds the capacity of adeno-associated viral (AAV) ...

journal_title：Gene therapy

authors： Gardner KL,Kearney JA,Edwards JD,Rafael-Fortney JA

更新日期：2006-05-01 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：: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：: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：: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：:The efficient delivery of genetic material to the developing fetal brain represents a powerful research tool and a means to supply therapy in a number of neonatal lethal neurological disorders. In this study, we have delivered vectors based upon adenovirus serotype 5 (Ad5) and adeno-associated virus (AAV) pseudotypes ...

journal_title：Gene therapy

authors： Rahim AA,Wong AM,Ahmadi S,Hoefer K,Buckley SM,Hughes DA,Nathwani AN,Baker AH,McVey JH,Cooper JD,Waddington SN

更新日期：2012-09-01 00:00:00

abstract：:The structure of 'stabilized plasmid-lipid particles' (SPLP) and their properties as systemic gene therapy vectors has been investigated. We show that SPLP can be visualized employing cryo-electron microscopy to be homogeneous particles of diameter 72 +/- 5 nm consisting of a lipid bilayer surrounding a core of plasmi...

journal_title：Gene therapy

authors： Tam P,Monck M,Lee D,Ludkovski O,Leng EC,Clow K,Stark H,Scherrer P,Graham RW,Cullis PR

更新日期：2000-11-01 00:00:00

abstract：:A persistent obstacle in the use of vector systems for gene therapy has been the inability to attain high-level expression of the target gene in primary cells in vivo. The MFG retroviral vector was designed to yield improved expression over the widely used N2 or LN vectors; however, the molecular basis for this effect...

journal_title：Gene therapy

authors： Krall WJ,Skelton DC,Yu XJ,Riviere I,Lehn P,Mulligan RC,Kohn DB

更新日期：1996-01-01 00:00:00

abstract：:The efficiency with which adenoviral vectors infect airway epithelial cells in vivo is unclear despite extensive preclinical and clinical studies. Our hypothesis is that gene transfer is limited by vector internalization which is mediated by binding of a fiber with a cellular receptor and the RGD motif of the penton b...

journal_title：Gene therapy

authors： Goldman M,Su Q,Wilson JM

更新日期：1996-09-01 00:00:00

abstract：:The prognosis of pancreatic adenocarcinoma is poor and current treatment ineffective. A novel treatment strategy is described here using a mouse model system for pancreatic cancer. Cells that have been genetically modified to express the cytochrome P450 2B1 enzyme are encapsulated in cellulose sulphate and implanted i...

journal_title：Gene therapy

authors： Löhr M,Müller P,Karle P,Stange J,Mitzner S,Jesnowski R,Nizze H,Nebe B,Liebe S,Salmons B,Günzburg WH

更新日期：1998-08-01 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：: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：: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：: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：:The proteasome is a multisubunit cytosolic protein complex responsible for degrading cytosolic proteins. Several studies have implicated its involvement in the processing of viral particles used for gene delivery, thereby limiting the efficiency of gene transfer. Peptide-based nonviral gene delivery systems are suffic...

journal_title：Gene therapy

authors： Kim J,Chen CP,Rice KG

更新日期：2005-11-01 00:00:00

abstract：:Complexes of DNA and cationic lipid offer potential advantages for gene transfer to airway epithelia. However, we found that application of DNA-lipid (DMRIE-DOPE) complexes to primary cultures of human ciliated airway epithelia or explants of rabbit trachea generated only low levels of gene transfer. In contrast, when...

journal_title：Gene therapy

authors： Fasbender A,Zabner J,Zeiher BG,Welsh MJ

更新日期：1997-11-01 00:00:00

abstract：:Intracardiac gene transfer and gene therapy have been investigated with different vector systems. Here we used adeno-associated virus (AAV) vectors to deliver either a reporter gene or a therapeutic gene into the heart of golden Syrian hamsters. The method of gene delivery was direct infusion of the AAV2 vectors into ...

journal_title：Gene therapy

authors： Li J,Wang D,Qian S,Chen Z,Zhu T,Xiao X

更新日期：2003-10-01 00:00:00

abstract：:Tissue-specific promoters for gene therapy are typically too big for adeno-associated virus (AAV) vectors; thus, the exploration of small effective non-viral regulatory elements is of particular interest. Wild-type AAV can specifically integrate into a region on human chromosome 19 termed AAVS1. Earlier work has deter...

journal_title：Gene therapy

authors： Li C,Hirsch M,Carter P,Asokan A,Zhou X,Wu Z,Samulski RJ

更新日期：2009-01-01 00:00:00