Gene doping detection: evaluation of approach for direct detection of gene transfer using erythropoietin as a model system.

abstract：:Inadequate antiprotease activity in the lungs due to alpha-1-antitrypsin (A1AT) deficiency is a factor of early-onset emphysema. We propose a new approach to gene therapy that involves the intratracheal delivery of macrophages expressing human A1AT (hA1AT). Recombinant adeno-associated virus (rAAV) plasmids encoding t...

journal_title：Gene therapy

authors： Zhang D,Wu M,Nelson DE,Pasula R,Martin WJ 2nd

更新日期：2003-12-01 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 progression of chronic renal diseases is considered as an irreversible process that eventually leads to end-stage renal failure characterized by extensive tissue fibrosis. At present, chronic renal fibrosis is incurable and the incidence of affected patients is on the rise worldwide. In this study, we demonstrate ...

journal_title：Gene therapy

authors： Yang J,Dai C,Liu Y

更新日期：2001-10-01 00:00:00

abstract：:Cultured keratinocyte allografts from unrelated donors can be readily grown as sheets in large-scale cell culture and have been used as an immediate skin cover for severely burned patients. Despite the absence of passenger leukocytes and the unlimited amount of material that can be obtained for permanent skin coverage...

journal_title：Gene therapy

authors： Mhashilkar AM,Doebis C,Seifert M,Busch A,Zani C,Soo Hoo J,Nagy M,Ritter T,Volk HD,Marasco WA

更新日期：2002-03-01 00:00:00

abstract：:The failure of pharmacological approaches to cure infection with the human immunodeficiency virus (HIV) has renewed the interest in gene-based therapies. Among the various strategies that are currently explored, the blockade of HIV entry into susceptible T cells and macrophages promises to be the most powerful interve...

journal_title：Gene therapy

authors： Schambach A,Schiedlmeier B,Kühlcke K,Verstegen M,Margison GP,Li Z,Kamino K,Bohne J,Alexandrov A,Hermann FG,von Laer D,Baum C

更新日期：2006-07-01 00:00:00

abstract：:Because periodontal ligament (PDL) cells are reported to contain progenitor or stem cell populations, they are considered a beneficial cell source for clinical periodontal regeneration. Both bone morphogenetic protein 4 (BMP4) and human telomerase reverse transcriptase (hTERT) have essential roles in the modulation of...

journal_title：Gene therapy

authors： Mi HW,Lee MC,Fu E,Chow LP,Lin CP

更新日期：2011-05-01 00:00:00

abstract：:Small interfering RNAs (siRNAs) are emerging as promising therapeutic tools. However, the widespread clinical application of such molecules as modulators of gene expression is still dependent on several aspects that limit their bioavailability. One of the most promising strategies to overcome the barriers faced by gen...

journal_title：Gene therapy

authors： Dharmapuri S,Peruzzi D,Marra E,Palombo F,Bett AJ,Bartz SR,Yong M,Ciliberto G,La Monica N,Buser CA,Toniatti C,Aurisicchio L

更新日期：2011-07-01 00:00:00

abstract：:Excessive activated T-cell proliferation was observed in vivo in one patient after an anti-CD19-chimeric antigen receptor (CAR) T-cell infusion. The patient, who had chemotherapy refractory and CD19+ diffuse large B-cell lymphoma (DLBCL), received an anti-CD19 CAR T-cell infusion following conditioning chemotherapy (f...

journal_title：Gene therapy

authors： Zhang WY,Liu Y,Wang Y,Nie J,Guo YL,Wang CM,Dai HR,Yang QM,Wu ZQ,Han WD

更新日期：2018-06-01 00:00:00

abstract：:Intravenous delivery of adenoviruses is the optimal route for many gene therapy applications. Once in the blood, coagulation factor X (FX) binds to the adenovirus capsid and protects the virion from natural antibody and classical complement-mediated neutralisation in mice. However, to date, no studies have examined th...

journal_title：Gene therapy

authors： Duffy MR,Doszpoly A,Turner G,Nicklin SA,Baker AH

更新日期：2016-07-01 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 have designed new vectors for the construction of recombinant adenoviruses containing expression cassettes in the E1 and/or E3 regions. Using a versatile set of restriction enzymes, the cassettes are cloned into small bacterial vectors and subsequently introduced into large plasmids containing the adenoviral sequen...

journal_title：Gene therapy

authors： Danthinne X,Werth E

更新日期：2000-01-01 00:00:00

abstract：:Inhibition of virus replication by means of RNA interference has been reported for several important human pathogens, including human immunodeficiency virus type 1 (HIV-1). RNA interference against these pathogens has been accomplished by introduction of virus-specific synthetic small interfering RNAs (siRNAs) or DNA ...

journal_title：Gene therapy

authors： Konstantinova P,de Vries W,Haasnoot J,ter Brake O,de Haan P,Berkhout B

更新日期：2006-10-01 00:00:00

abstract：:Adeno-associated virus (AAV) gene therapy constitutes a powerful tool for the treatment of neurodegenerative diseases. While AAVs are generally administered systemically to newborns in preclinical studies of neurological disorders, in adults the maturity of the blood-brain barrier (BBB) must be considered when selecti...

journal_title：Gene therapy

authors： Bey K,Ciron C,Dubreil L,Deniaud J,Ledevin M,Cristini J,Blouin V,Aubourg P,Colle MA

更新日期：2017-05-01 00:00:00

abstract：:To elucidate further the potential of a Semliki Forest virus (SFV) vector in vivo for gene therapy, we constructed a vector, SFV-IL12, to transfer murine IL-12 genes into tumors. A single intratumoral injection of established B16 murine melanoma with SFV-IL12 resulted in a significant inhibition of tumor growth, while...

journal_title：Gene therapy

authors： Asselin-Paturel C,Lassau N,Guinebretière JM,Zhang J,Gay F,Bex F,Hallez S,Leclere J,Peronneau P,Mami-Chouaib F,Chouaib S

更新日期：1999-04-01 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：: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：:The mouse is widely used as a model for DNA therapy and vaccination even though the efficiency of DNA delivery in higher mammals and humans is much less. The human APOBEC3 (A3) enzymes impact viral genomes by cytidine deamination, which introduces multiple uridine residues into single-stranded DNA, a process known as ...

journal_title：Gene therapy

authors： Kostrzak A,Henry M,Demoyen PL,Wain-Hobson S,Vartanian JP

更新日期：2015-01-01 00:00:00

abstract：:Retroviral vectors have become the primary tool for gene delivery into hematopoietic cells, including T lymphocytes. Lentiviral vectors offer an advantage over Moloney murine leukemia virus (MuLV) vectors because of their ability to translocate across an intact nuclear membrane and integrate into the genome of nonprol...

journal_title：Gene therapy

authors： Dardalhon V,Herpers B,Noraz N,Pflumio F,Guetard D,Leveau C,Dubart-Kupperschmitt A,Charneau P,Taylor N

更新日期：2001-02-01 00:00:00

abstract：:We recently showed that the human telomerase reverse transcriptase (hTERT) promoter induces tumor-specific Bax gene expression and selectively kills various human cancer cells both in vitro and in xenograft tumors. However, it remains unclear whether the hTERT promoter can be used to induce transgene expression in syn...

journal_title：Gene therapy

authors： Gu J,Andreeff M,Roth JA,Fang B

更新日期：2002-01-01 00:00:00

abstract：:We demonstrated previously that the additive-type recombinant Sendai virus (rSeV) is highly efficient for use in pulmonary gene transfer; however, rSeV exhibits inflammatory responses. To overcome this problem, we tested newly developed non-transmissible constructs, namely, temperature-sensitive F-deleted vector, rSeV...

journal_title：Gene therapy

authors： Tanaka S,Yonemitsu Y,Yoshida K,Okano S,Kondo H,Inoue M,Hasegawa M,Masumoto K,Suita S,Taguchi T,Sueishi K

更新日期：2007-07-01 00:00:00

abstract：:Intraperitoneal (i.p.) recurrence of cisplatin-refractory and p53 mutant ovarian cancer is a major clinical problem, despite surgery and chemotherapy. dl1520 (ONYX-015) is an E1B-55 kDa gene-deleted adenovirus engineered selectively to replicate in and destroy cancer cells lacking functional p53. However, a correlatio...

journal_title：Gene therapy

authors： Heise C,Ganly I,Kim YT,Sampson-Johannes A,Brown R,Kirn D

更新日期：2000-11-01 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：:Both atherosclerosis and arterial interventions induce oxidative stress mediated in part by nicotinamide adenine dinucleotide phosphate (NADPH) oxidases that have a pivotal role in the development of neointimal hyperplasia and restenosis. For small interfering RNA (siRNA) targeting of the NOX2 (Cybb) component of the ...

journal_title：Gene therapy

authors： Li JM,Newburger PE,Gounis MJ,Dargon P,Zhang X,Messina LM

更新日期：2010-10-01 00:00:00

abstract：:Breast cancer is the leading cancer diagnosed in women and the second leading cause of cancer-related deaths in women. Current limitations to standard chemotherapy in the clinic are extensively researched, including problems arising from repeated treatments with the same drugs. The phenomenon that cancer cells become ...

journal_title：Gene therapy

authors： Jones SK,Merkel OM

更新日期：2016-12-01 00:00:00

abstract：:Dendritic cell (DC)-based vaccines have been applied clinically in the setting of advanced-stage cancer. To date, the clinical efficacy of these vaccines has been limited, possibly owing to the impairment of transferred DC function in cancer-bearing patients. In this study, we examined the therapeutic efficacy of inte...

journal_title：Gene therapy

authors： Tatsumi T,Takehara T,Yamaguchi S,Sasakawa A,Miyagi T,Jinushi M,Sakamori R,Kohga K,Uemura A,Ohkawa K,Storkus WJ,Hayashi N

更新日期：2007-06-01 00:00:00

abstract：:The potential of gene therapy for treatment of lung disease remains unrealised. Early model systems often resulted in promising efficiency of gene transfer, only to prove irreproducible in the clinic. While problems such as induction of host immune responses and duration of expression also need to be addressed, it is ...

journal_title：Gene therapy

authors： McBride S,Rannie D,Harrison DJ

更新日期：2000-04-01 00:00:00

abstract：:Hepatic fibrosis is a common outcome of chronic liver diseases. In schistosomiasis, chronic parasite egg-induced granuloma formation can lead to fibrosis, which is immunologically characterized by the dominant Th2 response. Recently, it has been shown that gene therapy is an attractive approach for the treatment of he...

journal_title：Gene therapy

authors： Zhang LH,Pan JP,Yao HP,Sun WJ,Xia DJ,Wang QQ,He L,Wang J,Cao X

更新日期：2001-09-01 00:00:00

abstract：:First-generation adenoviral (Ad) vectors are frequently used vectors for experimental and clinical gene transfer. Earlier it has been shown that parallel overexpression of the cell cycle regulator p21(Waf1/Cip1) (p21) or antiapoptotic bcl-2 from a second vector reduces cytotoxicity and improves transgene expression. H...

journal_title：Gene therapy

authors： Schumacher A,Horvat S,Woischwill C,Wolff G,Witt C

更新日期：2009-04-01 00:00:00

abstract：:Reactive oxygen species (ROS) have been implicated in the pathogenesis of rheumatoid arthritis (RA), while antioxidant enzymes, such as extracellular superoxide dismutase (EC-SOD) and catalase, block radical-induced events. The present study tested if the ex vivo transfer of EC-SOD and catalase genes alone or in combi...

journal_title：Gene therapy

authors： Dai L,Claxson A,Marklund SL,Feakins R,Yousaf N,Chernajovsky Y,Winyard PG

更新日期：2003-04-01 00:00:00

abstract：:For certain gene therapy applications, the simultaneous delivery of multiple genes would allow for novel therapies. In the case of adeno-associated virus (AAV) vectors, the limited packaging capacity greatly restricts current methods of carrying multiple transgene cassettes. To address this issue, a recombinant AAV (r...

journal_title：Gene therapy

authors： Foti SB,Samulski RJ,McCown TJ

更新日期：2009-11-01 00:00:00