Retrovirus-mediated gene transfer into T cells: 95% transduction efficiency without further in vitro selection.

abstract：:Esophageal cancer is characterized by rapid clinical progression and poor prognosis, due to early-stage invasion of adjacent tissues and metastasis. Tissue factor pathway inhibitor-2 (TFPI-2) has been implicated as a metastasis-associated gene in many types of tumors. Here we describe the potential involvement of TFPI...

journal_title：Human gene therapy

authors： Ran Y,Pan J,Hu H,Zhou Z,Sun L,Peng L,Yu L,Sun L,Liu J,Yang Z

更新日期：2009-01-01 00:00:00

abstract：:Adeno-associated viral (AAV) vectors containing cone-specific promoters have rescued cone photoreceptor function in mouse and dog models of achromatopsia, but cone-specific promoters have not been optimized for use in primates. Using AAV vectors administered by subretinal injection, we evaluated a series of promoters ...

journal_title：Human gene therapy

authors： Ye GJ,Budzynski E,Sonnentag P,Nork TM,Sheibani N,Gurel Z,Boye SL,Peterson JJ,Boye SE,Hauswirth WW,Chulay JD

更新日期：2016-01-01 00:00:00

abstract：:Adoptive cellular therapy provides the promise of a potentially powerful general treatment for cancer. Although this is a complex and challenging field, there have been major advances in basic and translational research resulting in clinical trial activity that is now beginning to confirm this promise. However, these ...

journal_title：Human gene therapy

authors： Hawkins RE,Gilham DE,Debets R,Eshhar Z,Taylor N,Abken H,Schumacher TN,ATTACK Consortium

更新日期：2010-06-01 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：:Targeted delivery of intravenously administered genetically altered cells or stem cells is still in an early stage of investigation. We developed a method of delivering iron oxide (ferumoxide)-labeled mesenchymal stem cells (MSCs) to a targeted area in an animal model by applying an external magnet. Rats with or witho...

journal_title：Human gene therapy

authors： Arbab AS,Jordan EK,Wilson LB,Yocum GT,Lewis BK,Frank JA

更新日期：2004-04-01 00:00:00

abstract：:The epithelial-mesenchymal transition (EMT) has been recognized to occur during embryonic development, fibrosis, and tumor metastasis. Nuclear factor (NF)-κB plays a central role in mediating the inflammation and wound-healing responses during liver fibrogenesis. However, the involvement of NF-κB during EMT in liver c...

journal_title：Human gene therapy

authors： Kim KH,Lee WR,Kang YN,Chang YC,Park KK

更新日期：2014-08-01 00:00:00

abstract：:Engineering gene therapy vectors to modulate the immune response is an important goal. In this regard, costimulation of T cells is a critical determinant in immune activation. The costimulatory molecule CD40, expressed on antigen-presenting cells, is thought to interact with CD40 ligand (CD40L) expressed on activated ...

journal_title：Human gene therapy

authors： Sin JI,Kim JJ,Zhang D,Weiner DB

更新日期：2001-06-10 00:00:00

abstract：:Mutations in the gene encoding the peroxisomal ATP-binding cassette transporter (ABCD1) cause elevations in very long-chain fatty acids (VLCFAs) and the neurodegenerative disease adrenoleukodystrophy (ALD). In most adults, this manifests as the spinal cord axonopathy adrenomyeloneuropathy (AMN). A challenge in virus-b...

journal_title：Human gene therapy

authors： Gong Y,Berenson A,Laheji F,Gao G,Wang D,Ng C,Volak A,Kok R,Kreouzis V,Dijkstra IM,Kemp S,Maguire CA,Eichler F

更新日期：2019-05-01 00:00:00

abstract：:The recombinant adenoviral (Ad) vector is being considered as a cancer vaccine platform because it efficiently induces immune responses to tumor antigens by intradermal immunization. The aims of this study were to evaluate the potential toxicities and biodistribution after a single dose or six weekly intradermal doses...

journal_title：Human gene therapy

authors： Plog MS,Guyre CA,Roberts BL,Goldberg M,St George JA,Perricone MA

更新日期：2006-07-01 00:00:00

abstract：:Murine skeletal muscle cells transduced with foreign genes and tissue engineered in vitro into bioartificial muscles (BAMs) are capable of long-term delivery of soluble growth factors when implanted into syngeneic mice (Vandenburgh et al., 1996b). With the goal of developing a therapeutic cell-based protein delivery s...

journal_title：Human gene therapy

authors： Powell C,Shansky J,Del Tatto M,Forman DE,Hennessey J,Sullivan K,Zielinski BA,Vandenburgh HH

更新日期：1999-03-01 00:00:00

abstract：:Gene-modified lymphocytes have a potential role in the therapy of cancer, infectious diseases, and genetic disorders of the immune system. Current gene therapy protocols involving gene transfer into lymphocytes utilize retroviruses with amphotropic envelope proteins. However, transduction efficiencies in lymphocytes u...

journal_title：Human gene therapy

authors： Lam JS,Reeves ME,Cowherd R,Rosenberg SA,Hwu P

更新日期：1996-08-01 00:00:00

abstract：:Cell-based therapy for muscular dystrophies was initiated in humans after promising results obtained in murine models. Early trials failed to show substantial clinical benefit, sending researchers back to the bench, which led to the discovery of many hurdles as well as many new venues to optimize this therapeutic stra...

journal_title：Human gene therapy

authors： Negroni E,Bigot A,Butler-Browne GS,Trollet C,Mouly V

更新日期：2016-02-01 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：: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：: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：:To develop a potential gene therapy strategy for the treatment of hemophilia A, we constructed several retroviral vectors expressing a B-domain-deleted factor VIII (FVIII) cDNA. We confirmed previous reports that when the FVIII cDNA is inserted into a retroviral vector, the vector mRNA is decreased resulting in signif...

journal_title：Human gene therapy

authors： Chuah MK,VandenDriessche T,Morgan RA

更新日期：1995-11-01 00:00:00

abstract：:DNA expression vectors may be administered to patients like conventional medicines to have a finite and controlled duration of action. The clinical application of these medicines will require a precise understanding of the kinetics of the administered gene, the mRNA transcript, and the gene product. The apparent kinet...

journal_title：Human gene therapy

authors： Ledley TS,Ledley FD

更新日期：1994-06-01 00:00:00

abstract：:Peritoneal compartmentalization of advanced stage ovarian cancer provides a rational scenario for gene therapy strategies. Several groups are exploring intraperitoneal administration of adenoviral (Ad) vectors for this purpose. We examined in vitro gene transfer in the presence of ascites fluid from ovarian cancer pat...

journal_title：Human gene therapy

authors： Blackwell JL,Li H,Gomez-Navarro J,Dmitriev I,Krasnykh V,Richter CA,Shaw DR,Alvarez RD,Curiel DT,Strong TV

更新日期：2000-08-10 00:00:00

abstract：:Defined serum-free conditions have great conceptual advantages for the biological safety and standardization of clinical gene transfer into hematopoietic stem cells. In the only study reported to date, Sekhar et al. achieved low serum conditions by a complex concentration procedure of a retroviral supernatant initiall...

journal_title：Human gene therapy

authors： Glimm H,Flügge K,Möbest D,Hofmann VM,Postmus J,Henschler R,Lange W,Finke J,Kiem HP,Schulz G,Rosenthal F,Mertelsmann R,von Kalle C

更新日期：1998-04-10 00:00:00

abstract：:Liver ischemia-reperfusion (I/R) injury is a multifactorial process that affects graft function after liver transplantation. Inflammatory cytokines, such as tumor necrosis factor (TNF)-α, interleukin (IL)-6, IL-1β, and IL-18, have been shown to play key roles in the pathophysiology of liver I/R injury. Studies have in...

journal_title：Human gene therapy

authors： Zhu P,Duan L,Chen J,Xiong A,Xu Q,Zhang H,Zheng F,Tan Z,Gong F,Fang M

更新日期：2011-07-01 00:00:00

abstract：:Genetic immunization has been widely applied in efforts to find novel and efficient mechanisms of stimulating the immune response. An effective attack against viral pathogens or tumors often requires activation of T cell-mediated immunity and the generation of cytotoxic T cells. Intramuscular immunization with plasmid...

journal_title：Human gene therapy

authors： Hedley ML,Strominger JL,Urban RG

更新日期：1998-02-10 00:00:00

abstract：:Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene that result in the absence of functional protein. In the majority of cases these are out-of-frame deletions that disrupt the reading frame. Several attempts have been made to restore the dystrophin mRNA reading frame by modulation of pre-m...

journal_title：Human gene therapy

authors： Arechavala-Gomeza V,Graham IR,Popplewell LJ,Adams AM,Aartsma-Rus A,Kinali M,Morgan JE,van Deutekom JC,Wilton SD,Dickson G,Muntoni F

更新日期：2007-09-01 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：:Glycogen storage disease type II (GSDII) is a lysosomal storage disease caused by a deficiency in acid alpha-glucosidase (GAA), and leads to cardiorespiratory failure by the age of 2 years. In this study, we investigate the impact of anti-GAA antibody formation on cross-correction of the heart, diaphragm, and hind-lim...

journal_title：Human gene therapy

authors： Cresawn KO,Fraites TJ,Wasserfall C,Atkinson M,Lewis M,Porvasnik S,Liu C,Mah C,Byrne BJ

更新日期：2005-01-01 00:00:00

abstract：:Genome engineering has gone mainstream because of breakthroughs in defining and harnessing naturally occurring, customizable DNA recognition cursors (protein or RNA-guided). At present, most gene editing relies on these cursors to direct custom DNA endonucleases to a specific genomic sequence to induce a double-strand...

journal_title：Human gene therapy

authors： Martínez-Gálvez G,Ata H,Campbell JM,Ekker SC

更新日期：2016-06-01 00:00:00

abstract：:Three-vessel disease (TVD) is a severe coronary heart disease (CHD) with poor prognosis. Niemann-Pick C1-like 1 (NPC1L1) is a transporter protein for exogenous cholesterol absorption, and 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) is a rate-limiting enzyme for cholesterol synthesis. We aimed to investigat...

journal_title：Human gene therapy

authors： Zhao X,Li J,Tang X,Liu R,Xu J,Xu L,Jiang L,Huang K,Tian J,Feng X,Wu Y,Zhang Y,Wang D,Sun K,Xu B,Zhao W,Hui R,Gao R,Song L,Yuan J

更新日期：2021-01-22 00:00:00

abstract：:Human hematopoietic stem cells (HSCs) are poorly transduced by vectors based on adenovirus serotype 5 (Ad5). This is primarily due to the paucity of the coxsackievirus-Ad receptor on these cells. In an attempt to change the tropism of Ad5, we constructed a series of chimeric E1-deleted Ad5 vectors in which the shaft a...

journal_title：Human gene therapy

authors： Knaän-Shanzer S,Van Der Velde I,Havenga MJ,Lemckert AA,De Vries AA,Valerio D

更新日期：2001-11-01 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：:The practical application of gene transfer as a treatment for genetic diseases such as cystic fibrosis or hemophilia has been hindered, in part, by low efficiencies of vector delivery and transgene expression. We demonstrated that a feline immunodeficiency virus (FIV)-based lentiviral vector pseudotyped with the envel...

journal_title：Human gene therapy

authors： Sinn PL,Goreham-Voss JD,Arias AC,Hickey MA,Maury W,Chikkanna-Gowda CP,McCray PB Jr

更新日期：2007-12-01 00:00:00

abstract：:Following in vivo recombinant adeno-associated virus (rAAV)-based gene transfer, adaptive immune responses specific to the vector or the transgene product have emerged as a potential roadblock to successful clinical translation. The occurrence of such responses depends on several parameters, including the route of vec...

journal_title：Human gene therapy

authors： Gernoux G,Guilbaud M,Dubreil L,Larcher T,Babarit C,Ledevin M,Jaulin N,Planel P,Moullier P,Adjali O

更新日期：2015-01-01 00:00:00