Improved induction of immune tolerance to factor IX by hepatic AAV-8 gene transfer.

abstract：:With the aim of developing new gene transfer tools for treating CF with gene therapy, we have synthesized a novel family of molecules named cationic phosphonolipids. The most efficient among them were selected by in vitro screening to compare their activities in vivo in mouse lungs. We used a reporter gene whose activ...

journal_title：Human gene therapy

authors： Guillaume-Gable C,Floch V,Mercier B,Audrézet MP,Gobin E,Le Bolch G,Yaouanc JJ,Clément JC,des Abbayes H,Leroy JP,Morin V,Férec C

更新日期：1998-11-01 00:00:00

abstract：:PhD-trained biomedical scientists are moving into an increasingly diverse variety of careers within the sciences. However, graduate and postdoctoral training programs have historically focused on academic career preparation, and have not sufficiently prepared trainees for transitioning into other scientific careers. A...

journal_title：Human gene therapy

authors： Fuhrmann CN

更新日期：2016-11-01 00:00:00

abstract：:Mutations in the alpha-chain of lysosomal hexosaminidase (EC 3.2.1.52) underlie two distinct biochemical phenotypes known as variant B and variant B1 of G(M2) gangliosidosis. This paper shows that the transduction of human B1-type fibroblasts (producing catalytically inactive alpha-chains) with a retroviral vector enc...

journal_title：Human gene therapy

authors： Teixeira CA,Sena-Esteves M,Lopes L,Sá Miranda MC,Ribeiro MG

更新日期：2001-09-20 00:00:00

abstract：:Hyperoxia and ischemia-reperfusion cause profound lung cellular damage mediated, in part, by generation of oxygen radicals. We hypothesized that gene therapy can be used to overcome oxidant injury by augmenting intracellular antioxidant enzymes. Adult rats were injected intratracheally with an adenovirus (Ad) vector e...

journal_title：Human gene therapy

authors： Danel C,Erzurum SC,Prayssac P,Eissa NT,Crystal RG,Hervé P,Baudet B,Mazmanian M,Lemarchand P

更新日期：1998-07-01 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：:We report on an antitumor treatment involving electrogene therapy (EGT), a newly developed in vivo gene transfer method using electroporation. We carried out in vivo EGT in a subcutaneous model of CT26 colon carcinoma cells, using plasmid DNAs encoding interleukin 12 (IL-12) subunits. For this purpose, we developed tw...

journal_title：Human gene therapy

authors： Tamura T,Nishi T,Goto T,Takeshima H,Dev SB,Ushio Y,Sakata T

更新日期：2001-07-01 00:00:00

abstract：:Immunologically sensitized recipients present one of the most critical problems in clinical organ transplantation today, since preformed antibodies rapidly destroy donor tissue expressing specific MHC class I antigens (Ag). Therefore, sensitized patients are either unable to receive a compatible organ, or experience a...

journal_title：Human gene therapy

authors： Geissler EK,Graeb C,Tange S,Guba M,Jauch KW,Scherer MN

更新日期：2000-02-10 00:00:00

abstract：:Duchenne muscular dystrophy (DMD) and other inherited myopathies lead to progressive destruction of most skeletal muscles in the body, including those responsible for maintaining respiration. DMD is a fatal disorder caused by defects in the dystrophin gene. Recombinant adenovirus vectors (AdV) are considered a promisi...

journal_title：Human gene therapy

authors： Cho WK,Ebihara S,Nalbantoglu J,Gilbert R,Massie B,Holland P,Karpati G,Petrof BJ

更新日期：2000-03-20 00:00:00

abstract：:A major obstacle for the efficacy of cancer gene therapy is the need to transduce a high proportion of tumor cells with genes that directly or indirectly cause their death. During the formation of certain organs, cells compete among themselves to colonize the whole tissue. We reasoned that cell competition could be us...

journal_title：Human gene therapy

authors： Martinez-Quintanilla J,Cascallo M,Fillat C,Alemany R

更新日期：2009-07-01 00:00:00

abstract：:Traditionally, skeletal muscle and liver are the preferred target organs for gene transfer to supply a transgene product into the systemic circulation. In this respect, adipose tissue presents a number of attractive features. However, adipose tissue transduction in vivo has not been feasible by conventional methods. T...

journal_title：Human gene therapy

authors： Mizukami H,Mimuro J,Ogura T,Okada T,Urabe M,Kume A,Sakata Y,Ozawa K

更新日期：2006-09-01 00:00:00

abstract：:RNA interference (RNAi) is an evolutionarily conserved mechanism of posttranscriptional gene-specific silencing. For in vivo applications, RNAi has been hampered until recently by inefficient delivery methods and by the transient nature of the gene suppression. Lentiviral vectors (LVs) hold great promise for gene ther...

journal_title：Human gene therapy

authors： Van den Haute C,Eggermont K,Nuttin B,Debyser Z,Baekelandt V

更新日期：2003-12-10 00:00:00

abstract：:Recombinant adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great potential for human gene therapy. One major challenge for translation of promising research to clinical development is the manufacture of sufficient quantities of AAV vectors that meet stringent standards for p...

journal_title：Human gene therapy

authors： Wright JF

更新日期：2009-07-01 00:00:00

abstract：:Huntington's disease (HD) is a fatal neurodegenerative disease caused by a genetic expansion of the CAG repeat region in the huntingtin (HTT) gene. Studies in HD mouse models have shown that artificial miRNAs can reduce mutant HTT, but evidence for their effectiveness and safety in larger animals is lacking. HD transg...

journal_title：Human gene therapy

authors： Pfister EL,DiNardo N,Mondo E,Borel F,Conroy F,Fraser C,Gernoux G,Han X,Hu D,Johnson E,Kennington L,Liu P,Reid SJ,Sapp E,Vodicka P,Kuchel T,Morton AJ,Howland D,Moser R,Sena-Esteves M,Gao G,Mueller C,DiFiglia M

更新日期：2018-06-01 00:00:00

abstract：:Replication-deficient viral vectors are currently being used in gene transfer strategies to treat cancer cells. Unfortunately, viruses are limited in their ability to diffuse through tissue. This makes it virtually impossible to infect the majority of tumor cells in vivo and results in inadequate gene transfer. This p...

journal_title：Human gene therapy

authors： Han JS,Qian D,Wicha MS,Clarke MF

更新日期：1998-05-20 00:00:00

abstract：:Although replication-deficient adenoviruses can efficiently transfer genes to the salivary glands, the current vectors precipitate an immediate, transient decrease in salivary function. To study the cause of this salivary hypofunction, 10(6)-10(10) plaque-forming units (pfu) of the vector AdCMV beta gal were delivered...

journal_title：Human gene therapy

authors： Adesanya MR,Redman RS,Baum BJ,O'Connell BC

更新日期：1996-06-10 00:00:00

abstract：:Doxycycline (DOX) is widely used as a pharmacological agent and as an effector molecule in inducible gene expression systems. For most applications, it is important to determine whether the DOX concentration reaches the level required for optimal efficacy. We developed a sensitive bioassay for measuring the DOX concen...

journal_title：Human gene therapy

authors： Kleibeuker W,Zhou X,Centlivre M,Legrand N,Page M,Almond N,Berkhout B,Das AT

更新日期：2009-05-01 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：:Rat myoblast primary cultures were tested as a model for proinsulin synthesis and processing and unregulated insulin delivery for insulin-dependent diabetes mellitus (IDDM) gene therapy. Three human proinsulin cDNA constructs containing genetically engineered furin endoprotease cleavage sites between the B-chain and C...

journal_title：Human gene therapy

authors： Simonson GD,Groskreutz DJ,Gorman CM,MacDonald MJ

更新日期：1996-01-01 00:00:00

abstract：:Recombinant poxviruses expressing immunomodulatory molecules together with specific antigens represent powerful vaccines for cancer immunotherapy. Recently, we and others have demonstrated, in vitro and in vivo, that coexpression of CD80 and CD86 costimulatory molecules enhances the immunogenic capacity of a recombina...

journal_title：Human gene therapy

authors： Feder-Mengus C,Schultz-Thater E,Oertli D,Marti WR,Heberer M,Spagnoli GC,Zajac P

更新日期：2005-03-01 00:00:00

abstract：:The transfer of a drug resistance gene into hematopoietic cells is an approach being investigated to overcome the problem of myelosuppression produced by anticancer drugs. Chemotherapeutic agents are often given in combination in order to increase their effectiveness. Consequently, there is an advantage in designing v...

journal_title：Human gene therapy

authors： Beauséjour CM,Le NL,Létourneau S,Cournoyer D,Momparler RL

更新日期：1998-11-20 00:00:00

abstract：:Reporter gene-based molecular imaging can provide invaluable information on the fate of cellular therapies postimplantation. Integrating lentiviral vectors (ILVs) are commonly used for stably engineering cells; however, their potential for insertional mutagenesis poses a significant safety concern and barrier to wides...

journal_title：Human gene therapy

authors： Hamilton AM,Foster PJ,Ronald JA

更新日期：2018-10-01 00:00:00

abstract：:Development of multidrug resistance (MDR) is the major obstacle to successful cancer chemotherapy. We have developed Daudi human lymphoma cells that are 20-fold more resistant than the parent cell line to vincristine (VCR) by infecting cells with pHaMDR1/A retroviral vector (Daudi/MDR20). Three DNA sequences of anti-M...

journal_title：Human gene therapy

authors： Wang FS,Kobayashi H,Liang KW,Holland JF,Ohnuma T

更新日期：1999-05-01 00:00:00

abstract：:The utility of first-generation adenovirus vectors for long-term gene transfer in humans is limited by preexisting antiadenoviral immunity. We demonstrate here that new-generation high-capacity adenovirus vectors (HC-Ads) can efficiently transduce the brain and mediate stable transgene expression for at least 2 months...

journal_title：Human gene therapy

authors： Thomas CE,Schiedner G,Kochanek S,Castro MG,Lowenstein PR

更新日期：2001-05-01 00:00:00

abstract：:The therapeutic use of neurotrophic factors for neurodegenerative diseases is promising, however, optimal methods for continuous delivery of these substances to the human central nervous system (CNS) remains problematic. One approach would be to graft genetically engineered human cells that continuously secrete high l...

journal_title：Human gene therapy

authors： Lin Q,Cunningham LA,Epstein LG,Pechan PA,Short MP,Fleet C,Bohn MC

更新日期：1997-02-10 00:00:00

abstract：:Chronic inflammation in tibialis anterior muscles of mdx mice was produced by a single injection of a recombinant adenovirus vector (AV) expressing an immunogenic beta-galactosidase (beta-gal). In regions of intense beta-gal staining, mononuclear infiltrates abounded, and muscle fibers showed strong extrasynaptic utro...

journal_title：Human gene therapy

authors： Waheed I,Gilbert R,Nalbantoglu J,Guibinga GH,Petrof BJ,Karpati G

更新日期：2005-04-01 00:00:00

abstract：:The first report of in vivo gene delivery to the retina dates back to 1987 when a retroviral vector was injected intraocularly in newborn mice. Later came the observation that retinal cells could be successfully transduced using adenoviral and then adeno-associated and lentiviral vectors. By 2000, it had become clear ...

journal_title：Human gene therapy

authors： Auricchio A,Smith AJ,Ali RR

更新日期：2017-11-01 00:00:00

abstract：:Interleukin-12 (IL-12) is a cytokine that exhibits pleiotropic effects on lymphocytes and natural killer cells and has been shown to have promise for the immunotherapy of cancer. The combination of the immune costimulatory molecule B7.1 and IL-12 has been shown to be synergistic for T cell activation. By transfecting ...

journal_title：Human gene therapy

authors： Anderson R,Macdonald I,Corbett T,Hacking G,Lowdell MW,Prentice HG

更新日期：1997-06-10 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：:Retinal ganglion cells (RGCs) play a key role in the pathogenesis and development of glaucoma. The present study aims to investigate the underlying mechanism of long noncoding RNA growth arrest-specific transcript 5 (GAS5) in glaucoma development through regulating the apoptosis of RGCs. Rat models of chronic glaucoma...

journal_title：Human gene therapy

authors： Zhou RR,Li HB,You QS,Rong R,You ML,Xiong K,Huang JF,Xia XB,Ji D

更新日期：2019-12-01 00:00:00

abstract：:Immunotherapy with whole cell cancer vaccines has been tested in various tumor types. This study investigated the safety profile and antitumor activity of an allogeneic prostate carcinoma cell line, LNCaP, expressing recombinant human interleukin-2 and human interferon-gamma. Thirty HLA-A*0201-matched patients with pr...

journal_title：Human gene therapy

authors： Brill TH,Kübler HR,Pohla H,Buchner A,Fend F,Schuster T,van Randenborgh H,Paul R,Kummer T,Plank C,Eisele B,Breul J,Hartung R,Schendel DJ,Gansbacher B

更新日期：2009-12-01 00:00:00