Upregulation of Bag-1 by ex vivo gene transfer protects rat livers from ischemia/reperfusion injury.

abstract：:Three dogs with deficiency of the lysosomal enzyme alpha-L-iduronidase were treated by gene replacement therapy targeted at muscle. Direct intramuscular injections of plasmid encoding the alpha-L-iduronidase gene cDNA resulted in no detectable enzyme production, but may have resulted in immunologic sensitization to id...

journal_title：Human gene therapy

authors： Shull RM,Lu X,McEntee MF,Bright RM,Pepper KA,Kohn DB

更新日期：1996-08-20 00:00:00

abstract：:Pancreatic cancer is the fourth leading cause of cancer-related death in the United States, and even under optimal therapy these patients face a poor prognosis. Here we report a novel gene therapy-based strategy to battle this disease. We show that the majority of pancreatic tumors overexpress c-erb-B2, which therefor...

journal_title：Human gene therapy

authors： Liu X,Li J,Tian Y,Xu P,Chen X,Xie K,Qiu Z,Wang Y,Zhang D,Wolf F,Li C,Huang Q

更新日期：2010-02-01 00:00:00

abstract：:The optimal stem cell source for stem cell gene therapy has not been defined. Most gene transfer studies have used peripheral blood or marrow repopulating cells collected after administration of granulocyte colony-stimulating factor and stem cell factor (G-CSF/SCF). For clinical applications, however, growth factor ad...

journal_title：Human gene therapy

authors： Thomasson B,Peterson L,Thompson J,Goerner M,Kiem HP

更新日期：2003-11-20 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：: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：:Interleukin-12 (IL-12) is a heterodimeric cytokine that plays an important role in the development of cellular immunity. Clinical applications for this lymphokine include resolution of infectious disease, cancer immunotherapy, and boosting cellular immunity in AIDS patients. When using IL-12 and other cytokines therap...

journal_title：Human gene therapy

authors： Bramson J,Hitt M,Gallichan WS,Rosenthal KL,Gauldie J,Graham FL

更新日期：1996-02-10 00:00:00

abstract：:Current HIV-1 gene therapy approaches aim at stopping the viral life cycle at its earliest steps, such as entry or immediate postentry events. Among the most widely adopted strategies are CCR5 downregulation/knockout and the use of broadly neutralizing antibodies. However, the long-term efficacy and side effects are s...

journal_title：Human gene therapy

authors： Jung U,Urak K,Veillette M,Nepveu-Traversy MÉ,Pham QT,Hamel S,Rossi JJ,Berthoux L

更新日期：2015-10-01 00:00:00

abstract：:Artemis is a hairpin-opening endonuclease involved in nonhomologous end-joining and V(D)J recombination. Deficiency of Artemis results in radiation-sensitive severe combined immunodeficiency (SCID) characterized by complete absence of T and B cells due to an arrest at the receptor recombination stage. We have generate...

journal_title：Human gene therapy

authors： Multhaup M,Karlen AD,Swanson DL,Wilber A,Somia NV,Cowan MJ,McIvor RS

更新日期：2010-07-01 00:00:00

abstract：:Adeno-associated viral (AAV) vectors are becoming increasingly popular in basic research as well as in clinical gene therapy. Due to its exceptional resistance against physical and chemical stress, however, the increasing use of AAV in laboratories and clinics around the globe raises safety concerns. Proper decontamin...

journal_title：Human gene therapy

authors： Korte J,Mienert J,Hennigs JK,Körbelin J

更新日期：2020-11-06 00:00:00

abstract：:Recombinant adeno-associated virus (rAAV) is a prototypical gene therapy vector characterized by excellent safety profiles, wide host range, and the ability to transduce differentiated cells. Numerous rAAV-based vectors providing efficient and sustained expression of transgenes in target tissues have been developed fo...

journal_title：Human gene therapy

authors： Zolotukhin S

更新日期：2005-05-01 00:00:00

abstract：:Extension of in vivo nucleic acid transfection techniques and increased information about those transfection properties and side effects are urgently needed to advance biological research and drug therapy. Tissue pressure-mediated transfection, involving lightly pressing the target tissue after intravenous injection o...

journal_title：Human gene therapy

authors： Mukai H,Kawakami S,Kamiya Y,Ma F,Takahashi H,Satake K,Terao K,Kotera H,Yamashita F,Hashida M

更新日期：2009-10-01 00:00:00

abstract：:In vitro delivery of interferon-alpha (IFN-alpha) to cultured human monocytes by means of a replication-incompetent herpesvirus vector inhibits human immunodeficiency virus (HIV) replication. To explore the possibility of IFN-alpha gene delivery by vector-infected human monocytes, monocytes were isolated and the cultu...

journal_title：Human gene therapy

authors： Weir JP,Dacquel EJ,Aronovitz J

更新日期：1996-07-10 00:00:00

abstract：:Newcastle disease virus (NDV) is a naturally oncolytic virus that has been shown to be safe and effective for cancer therapy. Tumor virotherapy using NDV emerged in the 1950s and has advanced more recently by the increased availability of reverse genetics technology. In this study, we constructed a reverse genetics sy...

journal_title：Human gene therapy

authors： Wei D,Sun N,Nan G,Wang Y,Liu HQ,Peeters B,Chen ZN,Bian H

更新日期：2012-07-01 00:00:00

abstract：:Gene therapy studies in primates can provide important information regarding vector tropism, specific cellular expression, biodistribution, and safety prior to clinical trials. In this study, we report the assessment of transduction efficiency of recombinant adeno-associated virus (rAAV) vectors using human postmortem...

journal_title：Human gene therapy

authors： Fradot M,Busskamp V,Forster V,Cronin T,Léveillard T,Bennett J,Sahel JA,Roska B,Picaud S

更新日期：2011-05-01 00:00:00

abstract：:Advances in cell and gene therapy are opening up new avenues for regenerative medicine. Because of their acquired pluripotency, human induced pluripotent stem cells (hiPSCs) are a promising source of autologous cells for regenerative medicine. They show unlimited self-renewal while retaining the ability, in principle,...

journal_title：Human gene therapy

authors： Garate Z,Davis BR,Quintana-Bustamante O,Segovia JC

更新日期：2013-06-01 00:00:00

abstract：:Evaluation of the potential role of dendritic cells (DCs) as adjuvants for tumor vaccination has focused primarily on techniques that load DCs with peptide tumor antigens. Our aim has been to optimize the induction of antitumor immunity by enhancing the ability of DCs to present tumor-associated antigens endogenously ...

journal_title：Human gene therapy

authors： Wan Y,Bramson J,Carter R,Graham F,Gauldie J

更新日期：1997-07-20 00:00:00

abstract：:Adult T cell leukemia/lymphoma (ATL) is derived from CD4+ T cells and has a poor prognosis because of its resistance to chemotherapy. To evaluate the effectiveness of gene therapy for ATL, the effect of ganciclovir on ATL cell lines transfected with the thymidine kinase gene of herpes simplex virus type 1 (HSV-TK) was...

journal_title：Human gene therapy

authors： Obaru K,Fujii S,Matsushita S,Shimada T,Takatsuki K

更新日期：1996-12-01 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：:Previous studies have documented that the skin can be used as a bioreactor to produce proteins for systemic release to treat diseases. A gene-switch system has been developed that allows regulated expression of therapeutic genes. To determine whether this system could be used in the skin, we developed a transgenic mou...

journal_title：Human gene therapy

authors： Cao T,Tsai SY,O'Malley BW,Wang XJ,Roop DR

更新日期：2002-06-10 00:00:00

abstract：:Diabetes mellitus derives from either insulin deficiency (type I) or resistance (type II). Homozygous mutations in the insulin receptor (IR) gene cause the rare leprechaunism and Rabson-Mendenhall syndromes, severe forms of hyperinsulinemic insulin resistance for which no therapy is currently available. Systems have b...

journal_title：Human gene therapy

authors： Cotugno G,Pollock R,Formisano P,Linher K,Beguinot F,Auricchio A

更新日期：2004-11-01 00:00:00

abstract：:Gene therapy for heart diseases requires availability of an efficient vector for gene transfer into myocardium. Recombinant adenovirus expressing the Escherichia coli beta-galactosidase (beta-Gal) gene was shown to infect rat cardiocytes efficiently in vivo. However, a time course of gene expression showed that transg...

journal_title：Human gene therapy

authors： Gilgenkrantz H,Duboc D,Juillard V,Couton D,Pavirani A,Guillet JG,Briand P,Kahn A

更新日期：1995-10-01 00:00:00

abstract：:In vivo electroporation of plasmid DNA (DNA-EP) is an efficient and safe method for vaccines. It results in increased DNA uptake, enhances protein expression, and augments immune responses to the target antigen in a variety of species. To further improve the efficacy of DNA-EP, we evaluated small interfering RNA (siRN...

journal_title：Human gene therapy

authors： Dharmapuri S,Aurisicchio L,Biondo A,Welsh N,Ciliberto G,La Monica N

更新日期：2009-06-01 00:00:00

abstract：:Despite efforts toward improvements in retrovirus-mediated gene transfer, stable high-level expression of a therapeutic gene in human hematopoietic stem cells remains a great challenge. We have evaluated the efficiency of different viral long terminal repeats (LTRs) in long-term expression of a transgene in vivo, usin...

journal_title：Human gene therapy

authors： Kaneko S,Onodera M,Fujiki Y,Nagasawa T,Nakauchi H

更新日期：2001-01-01 00:00:00

abstract：:Blood vessels are among the easiest targets for gene therapy. However, no data are available about the safety and feasibility of intracoronary gene transfer in humans. We studied the safety and efficacy of catheter-mediated vascular endothelial growth factor (VEGF) plasmid/liposome (P/L) gene transfer in human coronar...

journal_title：Human gene therapy

authors： Laitinen M,Hartikainen J,Hiltunen MO,Eränen J,Kiviniemi M,Närvänen O,Mäkinen K,Manninen H,Syvänne M,Martin JF,Laakso M,Ylä-Herttuala S

更新日期：2000-01-20 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：:SV40 is an attractive potential vector with high-efficiency gene transfer into a wide variety of human tissues, including the bone marrow, a critical target organ for the cure of many diseases. In the present study, the three SV40 capsid proteins, VP1, VP2, and VP3, were produced in Spodoptera frugiperda (Sf9) insect ...

journal_title：Human gene therapy

authors： Sandalon Z,Dalyot-Herman N,Oppenheim AB,Oppenheim A

更新日期：1997-05-01 00:00:00

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：:Restoration of correct splicing of βIVS2-654-globin pre-mRNA was previously accomplished in erythroid cells from β-thalassemia/HbE patients by an engineered U7 small nuclear RNA (snRNA) that carried a sequence targeted to the cryptic branch point and an exonic splicing enhancer, U7.BP+623 snRNA. In this study, this ap...

journal_title：Human gene therapy

authors： d'Arqom A,Nualkaew T,Jearawiriyapaisarn N,Kole R,Svasti S

更新日期：2020-11-02 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：:Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a devastating disease caused by mutations in TYMP, which encodes thymidine phosphorylase (TP). In MNGIE patients, TP dysfunction results in systemic thymidine and deoxyuridine overload, which interferes with mitochondrial DNA replication. Preclinical stu...

journal_title：Human gene therapy

authors： Cabrera-Pérez R,Vila-Julià F,Hirano M,Mingozzi F,Torres-Torronteras J,Martí R

更新日期：2019-08-01 00:00:00