abstract：:Efficient gene delivery is a critical obstacle for gene therapy that must be overcome. Until current limits of gene delivery technology are solved, identification of systems with bystander effects is highly desirable. As an anticancer agent, radioactive iodine (131)I has minimal toxicity. The physical characteristics ...

journal_title：Gene therapy

authors： Mitrofanova E,Unfer R,Vahanian N,Link C

更新日期：2006-07-01 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：:Adeno-associated virus (AAV) derived vectors are considered highly eligible vehicles for human gene therapy. Not only do they possess many great potential for clinical applications due to their wide range of tissue targets but also their excellent preclinical safety profile makes them particularly suitable candidates ...

journal_title：Gene therapy

authors： Le Bec C,Douar AM

更新日期：2006-05-01 00:00:00

abstract：:Insulin-like growth factor-I (IGF-I) and its receptor (IGF-IR) are essential for normal ocular development and are expressed in numerous ocular cell types including lens epithelial cells, retinal pigment epithelial cells, Müller cells and endothelial cells. Endothelial cell proliferation is a common feature of prolife...

journal_title：Gene therapy

authors： Shaw LC,Pan H,Afzal A,Calzi SL,Spoerri PE,Sullivan SM,Grant MB

更新日期：2006-05-01 00:00:00

abstract：:Viral gene vectors often rely on packaging cell lines, which provide the necessary factors in trans for the formation of virus-like particles. Previously, we reported on a first-generation packaging cell line for gene vectors, which are based on the B-lymphotropic Epstein-Barr virus (EBV), a human gamma-herpesvirus. T...

journal_title：Gene therapy

authors： Hettich E,Janz A,Zeidler R,Pich D,Hellebrand E,Weissflog B,Moosmann A,Hammerschmidt W

更新日期：2006-05-01 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：: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：:We previously demonstrated that recombinant adeno-associated virus vectors based on serotype 2 (rAAV2) can direct transgene expression in salivary gland cells in vitro and in vivo. However, it is not known how other rAAV serotypes perform when infused into salivary glands. The capsids of serotypes 4 and 5 are distinct...

journal_title：Gene therapy

authors： Katano H,Kok MR,Cotrim AP,Yamano S,Schmidt M,Afione S,Baum BJ,Chiorini JA

更新日期：2006-04-01 00:00:00

abstract：:Over the last years, RNA interference (RNAi) has become a widely used technique that permits the knock-down, and hence functional analysis, of individual genes in vertebrate cells. However, the high failure rate of the RNA molecules used in RNAi experiments continues to be a problem. In this paper, I describe a set of...

journal_title：Gene therapy

authors： Cullen BR

更新日期：2006-03-01 00:00:00

abstract：:In 1998, two distinct and exciting scientific fields emerged which have profoundly shaped the current direction of biomedical research. The discovery of RNA interference (RNAi) and the derivation of human embryonic stem (ES) cells have yielded exciting new possibilities for researchers and clinicians alike. While fund...

journal_title：Gene therapy

authors： Heidersbach A,Gaspar-Maia A,McManus MT,Ramalho-Santos M

更新日期：2006-03-01 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：:Sendai virus (SeV) is able to transfect airway epithelial cells efficiently in vivo. However, as with other viral vectors, repeated administration leads to reduced gene expression. We have investigated the impact of inducing immunological tolerance to immunodominant T-cell epitopes on gene expression following repeate...

journal_title：Gene therapy

authors： Griesenbach U,Boyton RJ,Somerton L,Garcia SE,Ferrari S,Owaki T,Ya-Fen Z,Geddes DM,Hasegawa M,Altmann DM,Alton EW

更新日期：2006-03-01 00:00:00

abstract：:As soon as RNA interference (RNAi) was found to work in mammalian cells, research quickly focused on harnessing this powerful endogenous and specific mechanism of gene silencing for human therapy. RNAi uses small RNAs, less than 30 nucleotides in length, to suppress expression of genes with complementary sequences. Tw...

journal_title：Gene therapy

authors： Dykxhoorn DM,Palliser D,Lieberman J

更新日期：2006-03-01 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：: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：: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：: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：:Gene therapy is thought to be a promising method for the treatment of various diseases. One gene therapy strategy involves the manipulations on a process of formation of new vessels, commonly defined as angiogenesis. Angiogenic and antiangiogenic gene therapy is a new therapeutic approach to the treatment of cardiovas...

journal_title：Gene therapy

authors： Malecki M,Kolsut P,Proczka R

更新日期：2005-10-01 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：: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：: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：:Cell transplantation holds promise as a potential treatment for cardiac dysfunction. Our group has isolated populations of murine skeletal muscle-derived stem cells (MDSCs) that exhibit stem cell-like properties. Here, we investigated the fate of MDSCs after transplantation into the hearts of dystrophin-deficient mdx ...

journal_title：Gene therapy

authors： Payne TR,Oshima H,Sakai T,Ling Y,Gharaibeh B,Cummins J,Huard J

更新日期：2005-08-01 00:00:00

abstract：:More than one hundred different mutations in the gene encoding rhodopsin are associated with a group of retinal degenerations including retinitis pigmentosa, congenital stationary night blindness and retinitis punctata albescens. Given this large heterogeneity of mutations, it would be ideal to develop mutation-indepe...

journal_title：Gene therapy

authors： Cashman SM,Binkley EA,Kumar-Singh R

更新日期：2005-08-01 00:00:00

abstract：:With the identification of stem cell plasticity several years ago, multiple reports raised hopes that tissue repair by stem cell transplantation could be within reach in the near future. Krause et al reported that a single purified hematopoietic stem cell not only repopulated the bone marrow of a host animal, but also...

journal_title：Gene therapy

authors： Kashofer K,Bonnet D

更新日期：2005-08-01 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：:The development of efficient systems for in vivo gene transfer to the central nervous system (CNS) may provide a useful therapeutic strategy for the alleviation of several neurological disorders. In this study, we evaluated the feasibility of nonviral gene therapy to the CNS mediated by cationic liposomes. We present ...

journal_title：Gene therapy

authors： da Cruz MT,Cardoso AL,de Almeida LP,Simões S,de Lima MC

更新日期：2005-08-01 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：:Replication defective vectors derived from simple retroviruses or the more complex genomes of lentiviruses continue to offer the advantages of long-term expression, cell and tissue specific tropism, and large packaging capacity for the delivery of therapeutic genes. The occurrence of adverse events caused by insertion...

journal_title：Gene therapy

authors： Sinn PL,Sauter SL,McCray PB Jr

更新日期：2005-07-01 00:00:00

abstract：:The annual rate of kidney graft loss caused by chronic allograft nephropathy (CAN) has not improved over the past decade. Recent reports suggest that acute renal ischemia results in development of CAN. The goal of the present study was to assess the renoprotective potential and safety of hepatocyte growth factor (HGF)...

journal_title：Gene therapy

authors： Isaka Y,Yamada K,Takabatake Y,Mizui M,Miura-Tsujie M,Ichimaru N,Yazawa K,Utsugi R,Okuyama A,Hori M,Imai E,Takahara S

更新日期：2005-05-01 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：:Active disease of tuberculosis (TB) can be developed decades later by either a relapse of the initial infection (endogenous reactivation) or by an entrance of the secondary infection (exogenous reinfection), since the current chemotherapy cannot lead to complete elimination of tuberculosis. Although the immunotherapeu...

journal_title：Gene therapy

authors： Ha SJ,Jeon BY,Youn JI,Kim SC,Cho SN,Sung YC

更新日期：2005-04-01 00:00:00

abstract：:Overexpression of vascular endothelial growth factor (VEGF) and its cognate receptor KDR has been linked to a more aggressive phenotype of human prostate carcinomas. The importance of signal transduction through the VEGF receptor 2 is illustrated by use of soluble KDR, which binds to VEGF and sequesters this ligand be...

journal_title：Gene therapy

authors： Kaliberov SA,Kaliberova LN,Buchsbaum DJ

更新日期：2005-03-01 00:00:00

abstract：:The growth and metastasis of solid tumors relies on the activities of polypeptide growth factors to recruit stromal tissue and expand the tumor mass. Pleiotrophin (PTN) is a secreted growth factor with angiogenic activity that has been found to contribute to the growth and metastasis of tumors including melanoma. Here...

journal_title：Gene therapy

authors： Malerczyk C,Schulte AM,Czubayko F,Bellon L,Macejak D,Riegel AT,Wellstein A

更新日期：2005-02-01 00:00:00

abstract：:The accessibility of adipose tissue and its ability to secrete various bioactive molecules suggest that adipose cells may be attractive targets for gene therapy applications. Here, we report the use of highly defective herpes simplex virus (HSV) vectors as suitable gene transfer agents for adipose cells in culture and...

journal_title：Gene therapy

authors： Fradette J,Wolfe D,Goins WF,Huang S,Flanigan RM,Glorioso JC

更新日期：2005-01-01 00:00:00

abstract：:Current experimental gene therapy approaches for Parkinson's disease (PD) and dementia with Lewy bodies (DLB) include the use of viral vectors expressing antiapoptosis genes, neurotrophic factors and dopaminergic system enzymes. However, since increasing evidence favors a role for alpha-synuclein accumulation in the p...

journal_title：Gene therapy

authors： Hashimoto M,Rockenstein E,Mante M,Crews L,Bar-On P,Gage FH,Marr R,Masliah E

更新日期：2004-12-01 00:00:00

abstract：:RNA interference (RNAi) is an evolutionarily conserved process by which plants and animals protect their genomes utilizing small, double-stranded RNAs to degrade target RNAs in a sequence-specific manner. Post-transcriptional gene silencing by these moieties can lead to degradation of both cellular and viral RNAs. It ...

journal_title：Gene therapy

authors： Zhou N,Fang J,Mukhtar M,Acheampong E,Pomerantz RJ

更新日期：2004-12-01 00:00:00

abstract：:The concept and application of ethical principles in the context of medical research is changing rapidly. This is especially true for fast moving fields such as gene therapy, a relatively new but rapidly maturing field offering opportunities to influence health at a fundamental level, which may become a cornerstone of...

journal_title：Gene therapy

authors： Spink J,Geddes D

更新日期：2004-11-01 00:00:00

abstract：:Development of efficient short-term gene transfer technologies for embryonic stem (ES) cells is urgently needed for various existing and new ES cell-based research strategies. In this study, we present a highly efficient, nonviral non-DNA technology for genetic loading of mouse ES cells based on electroporation of def...

journal_title：Gene therapy

authors： Ponsaerts P,Brown JP,Van den Plas D,Van den Eeden L,Van Bockstaele DR,Jorens PG,Van Tendeloo VF,Merregaert J,Singh PB,Berneman ZN

更新日期：2004-11-01 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：:Gene therapy of cystic fibrosis (CF) lung disease needs highly efficient delivery and long-lasting complementation of the CFTR (cystic fibrosis transmembrane conductance regulator) gene into the respiratory epithelium. The development of lentiviral vectors has been a recent advance in the field of gene transfer and th...

journal_title：Gene therapy

authors： Copreni E,Penzo M,Carrabino S,Conese M

更新日期：2004-10-01 00:00:00