Retrovirus-mediated gene transfer of B7-1 and MHC class II converts a poorly immunogenic neuroblastoma into a highly immunogenic one.

abstract：:Adenoviruses (Ads) have shown great utility as vectors for the delivery of genes to mammalian cells, partly because of their ability to infect a wide range of different cell types independent of the replicative state of the cell. However, Ads do not transduce mature muscle efficiently because of low levels of the natu...

journal_title：Human gene therapy

authors： Bramson JL,Grinshtein N,Meulenbroek RA,Lunde J,Kottachchi D,Lorimer IA,Jasmin BJ,Parks RJ

更新日期：2004-02-01 00:00:00

abstract：:Recent marketing approval for genetically engineered hematopoietic stem and T cells bears witness to the substantial improvements in lentiviral vectors over the last two decades, but evaluations of the long-term efficacy and toxicity of gene and cell therapy products will, nevertheless, require further studies in nonh...

journal_title：Human gene therapy

authors： Sii-Felice K,Castillo Padilla J,Relouzat F,Cheuzeville J,Tantawet S,Maouche L,Le Grand R,Leboulch P,Payen E

更新日期：2019-10-01 00:00:00

abstract：:The enzyme hypoxanthine-guanine phosphoribosyltransferase (HGPRT) expressed by the parasite Trypanosoma brucei (Tb) can convert allopurinol, a purine analogue, to corresponding nucleotides with greater efficiency than its human homologue. We have developed a retroviral system that expresses the parasitic enzyme and te...

journal_title：Human gene therapy

authors： Trudeau C,Yuan S,Galipeau J,Benlimame N,Alaoui-Jamali MA,Batist G

更新日期：2001-09-01 00:00:00

abstract：:Accurate quantification of gene transfer (or gene correction) is a universal challenge in the field of gene therapy. In developing a clinical trial of lymphocyte gene therapy for Hunter syndrome (mucopolysaccharidosis type II), methods using Southern blot or automated DNA sequencing technology were employed, but found...

journal_title：Human gene therapy

authors： Becker K,Pan D,Whitley CB

更新日期：1999-10-10 00:00:00

abstract：:Human embryonic stem cells (hESC) and induced pluripotent stem cells (iPSC) offer great hope for in vitro modeling of Parkinson's disease (PD), as well as for designing cell-replacement therapies. To realize these opportunities, there is an urgent need to develop efficient protocols for the directed differentiation of...

journal_title：Human gene therapy

authors： Sánchez-Danés A,Consiglio A,Richaud Y,Rodríguez-Pizà I,Dehay B,Edel M,Bové J,Memo M,Vila M,Raya A,Izpisua Belmonte JC

更新日期：2012-01-01 00:00:00

abstract：:Adenovirus-based vectors comprise the most frequently used vector type in clinical studies to date. Both intense lab research and insights from the clinical trials reveal the importance of a comprehensive understanding of vector-host interactions. Especially for systemic intravenous adenovirus vector delivery, it is p...

journal_title：Human gene therapy

authors： Hagedorn C,Kreppel F

更新日期：2017-10-01 00:00:00

abstract：:To achieve effective gene therapy, it is necessary to selectively and efficiently transfect therapeutic gene into targeted cells. In this study, we developed a combination method using mannosylated lipoplexes, which show selectivity to antigen-presenting cells such as macrophages and dendritic cells, and bubble liposo...

journal_title：Human gene therapy

authors： Un K,Kawakami S,Suzuki R,Maruyama K,Yamashita F,Hashida M

更新日期：2010-01-01 00:00:00

abstract：:In the next decades, gene editing technologies are expected to be used in the treatment and prevention of human diseases. Yet, the future uses of gene editing in medicine are still unknown, including its applicability and effectiveness to the treatment and prevention of infectious diseases, cancer, and monogenic and p...

journal_title：Human gene therapy

authors： Rocha LFM,Braga LAM,Mota FB

更新日期：2020-08-01 00:00:00

abstract：:In previous studies we demonstrated that a modified human HSP70b promoter (HSE.70b) directs high levels of gene expression to tumor cells after mild hyperthermia treatment in the range of 41.5-44 degrees C. This transcriptional targeting system exhibits low basal activity at 37 degrees C, is highly induced (950-fold) ...

journal_title：Human gene therapy

authors： Brade AM,Szmitko P,Ngo D,Liu FF,Klamut HJ

更新日期：2003-03-20 00:00:00

abstract：:To evaluate the hypothesis that innate immune mechanisms play a major role in eliminating adenovirus (Ad) vectors from the lung, the fate of adenoviral genome of an Ad vector was quantified in the first 24 h after intratracheal administration of an Ad vector coding for beta-galactosidase (beta gal) to mice. Southern a...

journal_title：Human gene therapy

authors： Worgall S,Leopold PL,Wolff G,Ferris B,Van Roijen N,Crystal RG

更新日期：1997-09-20 00:00:00

abstract：:Administration of recombinant adenoviral (AdV) vectors to animals can lead to inflammatory and immune responses. For therapeutic indications in which repeated treatment is necessary, such as cystic fibrosis (CF), these responses can limit the therapeutic usefulness of the vector. In principle, the utility of the vecto...

journal_title：Human gene therapy

authors： Chu Q,St George JA,Lukason M,Cheng SH,Scheule RK,Eastman SJ

更新日期：2001-03-20 00:00:00

abstract：:We reported that SNV-derived retroviral vectors, which display single-chain antibodies on the viral surface, enable cell type-specific gene delivery into various human cells. In particular, the SNV cell type-specific gene delivery vector system appears to be well suited to transduce genes into cells of the human hemat...

journal_title：Human gene therapy

authors： Jiang A,Fisher H,Pomerantz RJ,Dornburg R

更新日期：1999-11-01 00:00:00

abstract：:Engineered measles virus (MV) strains deriving from the vaccine lineage represent a promising oncolytic platform and are currently being tested in phase I trials. In this study, we have demonstrated that MV strains genetically engineered to express the human sodium iodide symporter (NIS) have significant antitumor act...

journal_title：Human gene therapy

authors： Opyrchal M,Allen C,Iankov I,Aderca I,Schroeder M,Sarkaria J,Galanis E

更新日期：2012-04-01 00:00:00

abstract：:Hereditary tyrosinemia type 1 (HT1) is an autosomal recessive disorder caused by deficiency of fumarylacetoacetate hydrolase (FAH). It has been previously shown that ex vivo hepatocyte-directed gene therapy using an integrating lentiviral vector to replace the defective Fah gene can cure liver disease in small- and la...

journal_title：Human gene therapy

authors： VanLith C,Guthman R,Nicolas CT,Allen K,Du Z,Joo DJ,Nyberg SL,Lillegard JB,Hickey RD

更新日期：2018-11-01 00:00:00

abstract：:The gene therapy strategy using the hsvl-thymidine kinase gene (TK) and ganciclovir (GCV) injections that has been used for treating human glioblastomas has not been as effective as expected after the first animal experiments. A better understanding of the different steps involved in this treatment, like gene transfer...

journal_title：Human gene therapy

authors： Sturtz FG,Waddell K,Shulok J,Chen X,Caruso M,Sanson M,Snodgrass HR,Platika D

更新日期：1997-11-01 00:00:00

abstract：:The use of synthetic gene delivery systems in human gene transfer is hampered by poor transfection efficiencies, largely because of the inability of DNA to translocate across the nuclear pore complex. A means to overcome this barrier is to bind the DNA to nuclear localization signals (NLSs), which are recognized by sh...

journal_title：Human gene therapy

authors： Mesika A,Kiss V,Brumfeld V,Ghosh G,Reich Z

更新日期：2005-02-01 00:00:00

abstract：:At present, much more studies have focused on the role of microRNAs in osteoporosis, but the more specific role of microRNA-150-3p (miR-150-3p) in osteoporosis still needs full exploration. We aim at investigating the role of miR-150-3p in osteoporosis and at exploring the related mechanisms. Bone marrow mesenchymal s...

journal_title：Human gene therapy

authors： Qiu M,Zhai S,Fu Q,Liu D

更新日期：2021-01-22 00:00:00

abstract：:Gene transfer of the herpes simplex virus thymidine kinase (TK) gene associated with ganciclovir (GCV) treatment can lead to death of TK-expressing cells, and of neighboring TK- cells because of the bystander effect. Thus, a small proportion of TK+ cells in a tumor can lead to its complete regression after GCV treatme...

journal_title：Human gene therapy

authors： Qiao J,Black ME,Caruso M

更新日期：2000-07-20 00:00:00

abstract：:Deficiency of glycogen branching enzyme (GBE) causes glycogen storage disease type IV (GSD IV), which is characterized by the accumulation of a less branched, poorly soluble form of glycogen called polyglucosan (PG) in multiple tissues. This study evaluates the efficacy of gene therapy with an adeno-associated viral (...

journal_title：Human gene therapy

authors： Yi H,Zhang Q,Brooks ED,Yang C,Thurberg BL,Kishnani PS,Sun B

更新日期：2017-03-01 00:00:00

abstract：:A phase I clinical trial was conducted in which recombinant adenovirus containing the cystic fibrosis trans-membrane regulator (CFTR) (Ad2/CFTR) was administered by bronchoscopic instillation or aerosolization to the lungs of cystic fibrosis (CF) patients. In this paper, we evaluate the efficiency of Ad2/CFTR-mediated...

journal_title：Human gene therapy

authors： Perricone MA,Morris JE,Pavelka K,Plog MS,O'Sullivan BP,Joseph PM,Dorkin H,Lapey A,Balfour R,Meeker DP,Smith AE,Wadsworth SC,St George JA

更新日期：2001-07-20 00:00:00

abstract：:Efficient and homogeneous gene transfer to cardiac myocytes is a major target in myocardial gene therapy. The aim of this study was to determine the conditions permitting efficient, homogeneous, adenovirus-mediated gene transfer to cardiac myocytes, with a view to application during coronary artery catheterization. Ge...

journal_title：Human gene therapy

authors： Logeart D,Hatem SN,Rücker-Martin C,Chossat N,Névo N,Haddada H,Heimburger M,Perricaudet M,Mercadier JJ

更新日期：2000-05-01 00:00:00

abstract：:Oncolytic viruses (OV) are novel cancer gene therapies that are moving toward the forefront of modern medicines. However, their full therapeutic potential is hindered by the lack of convenient and reliable strategies to visualize and quantify OV growth kinetics and therapeutic efficacy in live cells. Here, we present ...

journal_title：Human gene therapy

authors： Quillien L,Top S,Kappler S,Redouté A,Dusetti N,Quentin-Froignant C,Lulka H,Camus C,Buscail L,Gallardo F,Bertagnoli S,Cordelier P

更新日期：2021-01-27 00:00:00

abstract：:Protein transduction domains (PTD), which can transport proteins or peptides across biological membranes, have been identified in several proteins of viral, invertebrate, and vertebrate origin. Here, we evaluate the immunological and biological consequences of including PTD in synthetic peptides and in DNA vaccines th...

journal_title：Human gene therapy

authors： Leifert JA,Lindencrona JA,Charo J,Whitton JL

更新日期：2001-10-10 00:00:00

abstract：:Metabolic myopathies are a diverse group of rare diseases in which impaired breakdown of stored energy leads to profound muscle dysfunction ranging from exercise intolerance to severe muscle wasting. Metabolic myopathies are largely caused by functional deficiency of a single gene and are generally subcategorized into...

journal_title：Human gene therapy

authors： Mah CS,Soustek MS,Todd AG,McCall A,Smith BK,Corti M,Falk DJ,Byrne BJ

更新日期：2013-11-01 00:00:00

abstract：:Hepatic stellate cells (HSCs) are the primary cell type responsible for liver fibrogenesis. Transforming growth factor beta 1 (TGF-β1) and platelet-derived growth factor (PDGF) are key profibrotic cytokines that regulate HSC activation and proliferation with functional convergence. Dual RNA interference against their ...

journal_title：Human gene therapy

authors： Jiang Y,Zhao Y,He F,Wang H

更新日期：2019-02-01 00:00:00

abstract：:Administration of plasmid/lipid complexes to the lung airways for the treatment of metastatic pulmonary diseases represents a new strategy of gene therapy. In this study we present evidence that intratracheal administration of a plasmid encoding murine IL-12 complexed with N-[1-(2,3-dioleyloxy)propyl)-N,N,N-trimethyla...

journal_title：Human gene therapy

authors： Blezinger P,Freimark BD,Matar M,Wilson E,Singhal A,Min W,Nordstrom JL,Pericle F

更新日期：1999-03-20 00:00:00

abstract：:This is an erratum of the published paper "Preclinical Evaluation of Chimeric Antigen Receptor-Modified T Cells Specific to Epithelial Cell Adhesion Molecule for Treating Colorectal Cancer". There are some errors in figure 6C and 7C in the article due to authors' mistakes when preparing the figures. Specifically, repr...

journal_title：Human gene therapy

authors： Wang W

更新日期：2019-08-01 00:00:00

abstract：:This study focuses on the design, construction, and evaluation of a chimeric promoter for gene therapy applications where it is desirable to have low-level basal expression of the newly transferred gene, which can be induced to higher levels of expression by the administration of pharmacologic agents that can be safel...

journal_title：Human gene therapy

authors： Suzuki M,Singh RN,Crystal RG

更新日期：1996-10-01 00:00:00

abstract：:Cell-based gene transfer using a stent platform would provide a significant advantage in terms of site-specific gene expression in the vasculature. The current study presents a novel stent design that allows stable in vivo transgene expression over a 4-week period in the vasculature. A mesh-stent coated with fibronect...

journal_title：Human gene therapy

authors： Panetta CJ,Miyauchi K,Berry D,Simari RD,Holmes DR,Schwartz RS,Caplice NM

更新日期：2002-02-10 00:00:00

abstract：:Extracellular vesicles (EVs) being released from two adjacent adeno-associated virus serotype 1 (AAV1)-producing 293T cells are shown by electron microscopy. We have shown that AAV vectors can associate with EVs and enter the media. Furthermore, we have recently reported that EV-associated AAV has robust gene delivery...

journal_title：Human gene therapy

authors： Fitzpatrick Z,György B,Skog J,Maguire CA

更新日期：2014-09-01 00:00:00