Inside out: optimization of lipid nanoparticle formulations for exterior complexation and in vivo delivery of saRNA.

abstract：:Varying degrees of metabolic abnormalities mediated by chronic inflammation are implicated in the chronic glomerular injuries associated with obesity. Interleukin (IL)-10, a pleiotropic cytokine, exerts anti-inflammatory effects in numerous biological settings. In the present study, we explored the biological benefits...

journal_title：Gene therapy

authors： Ogura M,Urabe M,Akimoto T,Onishi A,Ito C,Ito T,Tsukahara T,Mizukami H,Kume A,Muto S,Kusano E,Ozawa K

更新日期：2012-05-01 00:00:00

abstract：:As clinical gene therapy has progressed toward realizing its potential, concern over misuse of the technology to enhance performance in athletes is growing. Although 'gene doping' is banned by the World Anti-Doping Agency, its detection remains a major challenge. In this study, we developed a methodology for direct de...

journal_title：Gene therapy

authors： Baoutina A,Coldham T,Bains GS,Emslie KR

更新日期：2010-08-01 00:00:00

abstract：:The current therapies to treat hepatitis B virus (HBV) infection are limited. Recently, clustered regularly interspaced short palindromic repeat (CRISPR) systems, originally identified in bacteria and archaea, have been found to consist of an RNA-based adaptive immune system that degrades complimentary sequences of in...

journal_title：Gene therapy

authors： Zhen S,Hua L,Liu YH,Gao LC,Fu J,Wan DY,Dong LH,Song HF,Gao X

更新日期：2015-05-01 00:00:00

abstract：:Glioblastoma multiforme (GBM) is one of the most formidable brain tumors with a mean survival period of approximately 12 months. To date, a combination of radiotherapy and chemotherapy with an oral alkylating agent, temozolomide (TMZ), has been used as first-line therapy for glioma. However, the efficacy of chemothera...

journal_title：Gene therapy

authors： Kato T,Natsume A,Toda H,Iwamizu H,Sugita T,Hachisu R,Watanabe R,Yuki K,Motomura K,Bankiewicz K,Wakabayashi T

更新日期：2010-11-01 00:00:00

abstract：:We explored the possibility of using a genetic approach to inhibit integrin-mediated endothelial cell adhesion and survival. We constructed recombinant adenoviruses (Ads) expressing chimeric proteins consisting of the cytoplasmic and transmembrane domains of integrin beta1 (CH1), beta3 (CH3) or the beta1 transmembrane...

journal_title：Gene therapy

authors： Oguey D,George PW,Rüegg C

更新日期：2000-08-01 00:00:00

abstract：:Choroidal neovascularization (CNV) leads to loss of vision in age-related macular degeneration (AMD), the leading cause of blindness in adult population over 50 years old. In this study, we developed intravenously administered, nanoparticulate, targeted nonviral retinal gene delivery systems for the management of CNV....

journal_title：Gene therapy

authors： Singh SR,Grossniklaus HE,Kang SJ,Edelhauser HF,Ambati BK,Kompella UB

更新日期：2009-05-01 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：: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：:Therapeutic RNA interference (RNAi) has emerged as a promising approach for the treatment of many incurable diseases, including cancer, infectious disease or neurodegenerative disorders. Demonstration of efficacy and safety in animal models is necessary before planning human application. Our group and others have prev...

journal_title：Gene therapy

authors： Martin JN,Wolken N,Brown T,Dauer WT,Ehrlich ME,Gonzalez-Alegre P

更新日期：2011-07-01 00:00:00

abstract：:Polynucleotide immunization has been employed as a means of inducing immune responses through the introduction of antigen-encoding DNA. While immunization against specific tumor antigens may be achieved through this strategy, various candidate tumor antigens may not be approached via DNA-based vaccines as they represe...

journal_title：Gene therapy

authors： Strong TV,Hampton TA,Louro I,Bilbao G,Conry RM,Curiel DT

更新日期：1997-06-01 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：: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：:The corticospinal tract (CST) is extensively used as a model system for assessing potential therapies to enhance neuronal regeneration and functional recovery following spinal cord injury (SCI). However, efficient transduction of the CST is challenging and remains to be optimised. Recombinant adeno-associated viral (A...

journal_title：Gene therapy

authors： Hutson TH,Verhaagen J,Yáñez-Muñoz RJ,Moon LD

更新日期：2012-01-01 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：:CC-chemokine receptor (CCR)-5 is the principal coreceptor for the entry of macrophage (M)-tropic HIV-1 viruses into a cell, while CXC-chemokine receptor (CXCR)-4 is the principal coreceptor for T cell line (T)-tropic HIV-1. In this study, we utilized a novel intracellular chemokine (intrakine) strategy to co-inactivat...

journal_title：Gene therapy

authors： Bai X,Chen JD,Yang AG,Torti F,Chen SY

更新日期：1998-07-01 00:00:00

abstract：:Multiple sclerosis (MS) is an immune-mediated disease of the central nervous system (CNS), where suspected autoimmune attack causes nerve demyelination and progressive neurodegeneration and should benefit from both anti-inflammatory and neuroprotective strategies. Although neuroprotection strategies are relatively une...

journal_title：Gene therapy

authors： Baker D,Hankey DJ

更新日期：2003-05-01 00:00:00

abstract：:We tested the longevity of gene expression provided by autonomously replicating vectors. The vectors contain segments of human genomic DNA that provide efficient replication initiation and sequences derived from Epstein-Barr virus that provide nuclear retention. In order to monitor gene expression, the vectors also ca...

journal_title：Gene therapy

authors： Wohlgemuth JG,Kang SH,Bulboaca GH,Nawotka KA,Calos MP

更新日期：1996-06-01 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

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：:A stable packaging cell line (Vero/BC-F) constitutively expressing fusion (F) protein of the human parainfluenza virus type 2 (hPIV2) was established for production of the F-defective and single round-infectious hPIV2 vector in a strategy for recombinant vaccine development. The F gene expression has not evoked cytost...

journal_title：Gene therapy

authors： Ohtsuka J,Fukumura M,Tsurudome M,Hara K,Nishio M,Kawano M,Nosaka T

更新日期：2014-08-01 00:00:00

abstract：:Retroviral vectors are effective shuttle systems by introducing therapeutically relevant genes stably into the genome of proliferating cells. The majority of vectors applied for research or clinical applications use neomycin for cell selection and identification. To circumvent the time consuming and potentially toxic ...

journal_title：Gene therapy

authors： Machl AW,Planitzer S,Kubbies M

更新日期：1997-04-01 00:00:00

abstract：:A replication-incompetent adenoviral vector encoding the heavy chain C-fragment (H(C)50) of botulinum neurotoxin type C (BoNT/C) was evaluated as a mucosal vaccine against botulism in a mouse model. Single intranasal inoculation of the adenoviral vector elicited a high level of H(C)50-specific IgG, IgG1 and IgG2a in s...

journal_title：Gene therapy

authors： Xu Q,Pichichero ME,Simpson LL,Elias M,Smith LA,Zeng M

更新日期：2009-03-01 00:00:00

abstract：:The bystander effect (BSE) is an interesting and important property of the herpes thymidine kinase/ganciclovir (hTK/GCV) system of gene therapy for cancer. With the BSE, not only are the hTK expressing cells killed upon ganciclovir (GCV) exposure but also neighboring wild-type tumor cells. On testing a large number of...

journal_title：Gene therapy

authors： Touraine RL,Ishii-Morita H,Ramsey WJ,Blaese RM

更新日期：1998-12-01 00:00:00

abstract：:Several of the current techniques for transfer of both oligonucleotide and plasmid DNA into the myocardium are impaired by low efficiency and toxicity. To improve gene transfer techniques, especially into the whole heart, a gene transfer method involving liposome in conjunction with a viral envelope (HVJ-liposome) was...

journal_title：Gene therapy

authors： Sawa Y,Kaneda Y,Bai HZ,Suzuki K,Fujimoto J,Morishita R,Matsuda H

更新日期：1998-11-01 00:00:00

abstract：:A recombinant adeno-associated virus (rAAV) vector was used to overexpress the anti-apoptotic Bcl-2-family protein, BCL-w, in rat brain. Three weeks after injecting the vector into cerebral cortex and striatum on one side, temporary focal ischemia was induced by occlusion of the ipsilateral middle cerebral artery for ...

journal_title：Gene therapy

authors： Sun Y,Jin K,Clark KR,Peel A,Mao XO,Chang Q,Simon RP,Greenberg DA

更新日期：2003-01-01 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：:Human immunodeficiency virus (HIV) infection ultimately leads to the destruction of the CD4+ lymphocyte subset and the onset of AIDS. In recent years, several gene therapy procedures making use of retroviral vectors that selectively target HIV susceptible cells have been proposed in order to interfere with HIV product...

journal_title：Gene therapy

authors： Lodge R,Subbramanian RA,Forget J,Lemay G,Cohen EA

更新日期：1998-05-01 00:00:00

abstract：:The E1 deleted adenoviral vectors are efficient at gene transfer to cells in culture or in animals. However, their use is limited because of an immune-mediated loss of transduced cells. This immune response is believed to result from low-level production of viral antigens from these vectors after gene transfer. The ea...

journal_title：Gene therapy

authors： Scaria A,Curiel DT,Kay MA

更新日期：1995-06-01 00:00:00

abstract：:Soluble receptors to vascular endothelial growth factor (VEGF) can inhibit its angiogenic effect. Since angiogenesis is involved in wound repair, we hypothesized that adenovirus-mediated gene transfer of a soluble form of VEGF receptor 2 (Flk-1) would attenuate wound healing in mice. C57Bl/6J and genetically diabetic ...

journal_title：Gene therapy

authors： Jacobi J,Tam BY,Sundram U,von Degenfeld G,Blau HM,Kuo CJ,Cooke JP

更新日期：2004-02-01 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