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：: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：: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：: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：: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：: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：:Glycogen storage disease type II (GSD-II) is a lethal, autosomal recessive metabolic myopathy caused by a lack of acid-alpha-glucosidase (GAA) activity in the cardiac and skeletal muscles. Absence of adequate intralysosomal GAA activity results in massive amounts of glycogen accumulation in multiple muscle groups, res...

journal_title：Human gene therapy

authors： Ding EY,Hodges BL,Hu H,McVie-Wylie AJ,Serra D,Migone FK,Pressley D,Chen YT,Amalfitano A

更新日期：2001-05-20 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：: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：:Umbilical cord blood (CB) from the early gestational human fetus is recognized as a rich source of hematopoietic stem cells. To examine the value of fetal CB for gene therapy of inborn immunohematopoietic disorders, we tested the feasibility of genetic modification of CD34(+) cells from CB at weeks 24 to 34 of pregnan...

journal_title：Human gene therapy

authors： Luther-Wyrsch A,Costello E,Thali M,Buetti E,Nissen C,Surbek D,Holzgreve W,Gratwohl A,Tichelli A,Wodnar-Filipowicz A

更新日期：2001-03-01 00:00:00

abstract：:A human immunodeficiency virus type 1 (HIV-1)-based retroviral vector pseudotyped with HIV envelope containing the herpes simplex virus-thymidine kinase (HSV-TK) gene under the control of the HIV LTR promoter (pHXTKN) was constructed and stably transferred into human CD4(+) H9, CEM, and U937 cells. RNase protection as...

journal_title：Human gene therapy

authors： Miyake K,Iijima O,Suzuki N,Matsukura M,Shimada T

更新日期：2001-02-10 00:00:00

abstract：:Retroviral vectors encoding glucose-responsive promoters driving furin expression may provide an amplified, glucose-regulated secretion of insulin. We constructed LhI*TFSN virus to encode a glucose-regulatable transforming growth factor alpha promoter controlling furin expression with a viral LTR promoter driving cons...

journal_title：Human gene therapy

authors： Barry SC,Ramesh N,Lejnieks D,Simonson WT,Kemper L,Lernmark A,Osborne WR

更新日期：2001-01-20 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：:The in vitro genetic manipulation of dendritic cells (DCs) for the expression of foreign proteins or peptides will assist in the development of immunotherapeutic approaches to treat cancer, immunological disorders, and/or infectious diseases. Reports have shown the expansion and differentiation of CD34(+) progenitor c...

journal_title：Human gene therapy

authors： Evans JT,Cravens P,Lipsky PE,Garcia JV

更新日期：2000-12-10 00:00:00

abstract：:Janus kinase 3 (JAK3) is an essential component of cytokine receptor signal transduction pathways required for normal lymphocyte development and function. JAK3 deficiency in both mice and humans results in severe combined immunodeficiency (SCID) and increased susceptibility to opportunistic infections. We have previou...

journal_title：Human gene therapy

authors： Bunting KD,Lu T,Kelly PF,Sorrentino BP

更新日期：2000-11-20 00:00:00

abstract：:Pulmonary edema is cleared via active Na(+) transport by alveolar epithelial Na(+)/K(+)-ATPases and Na(+) channels. Rats exposed to acute hyperoxia have a high mortality rate, decreased Na(+)/K(+)-ATPase function, and decreased alveolar fluid clearance (AFC). We hypothesized that Na(+)/K(+)-ATPase subunit gene overexp...

journal_title：Human gene therapy

authors： Factor P,Dumasius V,Saldias F,Brown LA,Sznajder JI

更新日期：2000-11-01 00:00:00

abstract：:The combination of immunization strategies with gene therapy methods constitutes a powerful tool for the purpose of genetic immunization. The cutaneous microenvironment, rich in professional antigen-presenting cells and accessory cells capable of initiating and controlling the intensity of specific immune responses, m...

journal_title：Human gene therapy

authors： Larregina AT,Falo LD Jr

更新日期：2000-11-01 00:00:00

abstract：:Recombinant adeno-associated virus (AAV) holds much promise for human gene therapy. While evidence indicates that AAV mediates long-term gene transfer in several different tissues, difficulty in preparing and purifying this viral vector in large quantities remains a major obstacle for evaluating AAV vectors in clinica...

journal_title：Human gene therapy

authors： Gao G,Qu G,Burnham MS,Huang J,Chirmule N,Joshi B,Yu QC,Marsh JA,Conceicao CM,Wilson JM

更新日期：2000-10-10 00:00:00

abstract：:The efficiency of gene therapy strategies against cancer is limited by the poor distribution of the vectors in the malignant tissues. To solve this problem, a new generation of tumor-specific, conditionally replicative adenoviruses is being developed. To direct the replication of the virus to breast cancer, we have co...

journal_title：Human gene therapy

authors： Hernandez-Alcoceba R,Pihalja M,Wicha MS,Clarke MF

更新日期：2000-09-20 00:00:00

abstract：:Based on the K8/JTS-1-mediated transfection technique, we developed an in vivo protocol for an efficient transfer of plasmid DNA to ocular cells. As determined with condensed plasmids containing reporter genes for either beta-galactosidase (pcDNA-lacZ) or enhanced green fluorescent protein (pREP-EGFP), the immortalize...

journal_title：Human gene therapy

authors： Neuner-Jehle M,Berghe LV,Bonnel S,Uteza Y,Benmeziane F,Rouillot JS,Marchant D,Kobetz A,Dufier JL,Menasche M,Abitbol M

更新日期：2000-09-01 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：: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：:Subcutaneous vaccination therapy with glioma cells, which are retrovirally transduced to secrete granulocyte-macrophage colony-stimulating factor (GM-CSF), has previously proven effective in C57BL/6 mice harboring intracerebral GL261 gliomas. However, clinical ex vivo gene therapy for human gliomas would be difficult,...

journal_title：Human gene therapy

authors： Herrlinger U,Jacobs A,Quinones A,Woiciechowsky C,Sena-Esteves M,Rainov NG,Fraefel C,Breakefield XO

更新日期：2000-07-01 00:00:00

abstract：:T lymphocytes, regardless of their specificity, are considered key targets for genetic modification in the treatment of inherited or acquired human diseases. In this study, we generated Lewis T cell lines specific for Dark Agouti rat alloantigens and tested the potential of allospecific T lymphocytes as carriers of ge...

journal_title：Human gene therapy

authors： Hammer MH,Flügel A,Seifert M,Lehmann M,Brandt C,Volk HD,Ritter T

更新日期：2000-06-10 00:00:00

abstract：:This study was designed to retrovirally transduce T cells by a protocol that would be simple, short, cost effective, applicable for clinical use, and efficient enough to avoid further selection of transduced T cells. Because retrovirally mediated infection is depending on the cell cycle, we first optimized the conditi...

journal_title：Human gene therapy

authors： Movassagh M,Boyer O,Burland MC,Leclercq V,Klatzmann D,Lemoine FM

更新日期：2000-05-20 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：:Replication-deficient adenovirus vector (Ad) is one of the most efficient gene transfer vehicles for human gene therapy. However, Ad is antigenic, known to evoke prominent inflammatory responses in vivo, and there are concerns that using Ad in patients with immune-mediated disorders (allergy and autoimmune diseases) m...

journal_title：Human gene therapy

authors： Suzuki M,Suzuki S,Yamamoto N,Komatsu S,Inoue S,Hashiba T,Nishikawa M,Ishigatsubo Y

更新日期：2000-04-10 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：:The central nervous system (CNS) is a predominant site of involvement in several lysosomal storage diseases (LSDs); and for many patients, these diseases are diagnosed only after the onset of symptoms related to the progressive accumulation of macromolecules within lysosomes. The mucopolysaccharidosis type VII (MPS VI...

journal_title：Human gene therapy

authors： Sferra TJ,Qu G,McNeely D,Rennard R,Clark KR,Lo WD,Johnson PR

更新日期：2000-03-01 00:00:00

abstract：:Recombinant adenoviruses have great potential as gene delivery systems because of their ability to infect a wide range of target cells. However, systemic delivery of viral vectors to tissues other than liver and spleen has been inefficient because of the rapid clearance of the circulating virus by the liver. In the pr...

journal_title：Human gene therapy

authors： Ye X,Jerebtsova M,Ray PE

更新日期：2000-03-01 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：: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：:Replication-competent adenoviruses may provide a highly efficient means of delivering therapeutic genes to tumors. Previously, we evaluated in vitro a replication-competent adenovirus (Ad5-CD/TKrep) containing a cytosine deaminase (CD)/herpes simplex type 1 thymidine kinase (HSV-1 TK) fusion gene that allows lytic vir...

journal_title：Human gene therapy

authors： Rogulski KR,Wing MS,Paielli DL,Gilbert JD,Kim JH,Freytag SO

更新日期：2000-01-01 00:00:00

abstract：:Targeted vectors provide a number of advantages for systemic and local gene delivery strategies. Several groups have investigated the utility of using various ligands to alter the tropism of adenovirus (Ad) vectors. We have previously demonstrated that fibroblast growth factor (FGF) ligands can specifically target DNA...

journal_title：Human gene therapy

authors： Printz MA,Gonzalez AM,Cunningham M,Gu DL,Ong M,Pierce GF,Aukerman SL

更新日期：2000-01-01 00:00:00

abstract：:Fabry disease, caused by a deficiency of lysosomal enzyme alpha-galactosidase A (alpha-gal A), is one of the inherited disorders potentially treatable by gene transfer to hematopoietic stem cells. In this study, a high-titer amphotropic retroviral producer cell line, MFG-alpha-gal A, was established. CD34+ cells from ...

journal_title：Human gene therapy

authors： Takiyama N,Dunigan JT,Vallor MJ,Kase R,Sakuraba H,Barranger JA

更新日期：1999-12-10 00:00:00

abstract：:An E1-, E2a-, E3-deleted adenoviral vector (Av3H82) encoding an epitope-tagged B domain-deleted human factor VIII cDNA (flagged FVIII) was evaluated in nonhuman primates. Twelve cynomolgus monkeys received intravenous administration of Av3H82; 6 monkeys received 6 x 10(11) particles/kg and another 6 received 3 x 10(12...

journal_title：Human gene therapy

authors： Brann T,Kayda D,Lyons RM,Shirley P,Roy S,Kaleko M,Smith T

更新日期：1999-12-10 00:00:00

abstract：:DNA vaccination is an attractive approach for tumor immunotherapy because of its stability and simplicity of delivery. Advances demonstrate that helper T cell responses play a critical role in initiating immune responses. The aim of the current study is to test whether targeting HPV-16 E7 to the endosomal/lysosomal co...

journal_title：Human gene therapy

authors： Ji H,Wang TL,Chen CH,Pai SI,Hung CF,Lin KY,Kurman RJ,Pardoll DM,Wu TC

更新日期：1999-11-20 00:00:00

abstract：:Deoxyribozymes, or DNA enzymes (DNAzymes), are novel nucleic acids that have the ability to bind to specific sequences of RNA, and to cleave the target site catalytically. DNAzymes are smaller and more efficient enzymatically than ribozymes (RZs), which are catalytic nucleic acids synthesized from ribonucleotides. We ...

journal_title：Human gene therapy

authors： Wu Y,Yu L,McMahon R,Rossi JJ,Forman SJ,Snyder DS

更新日期：1999-11-20 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：:An RNA melanoma vaccine was investigated to induce protective immunity in a mouse-melanoma model. LacZ mRNA was synthesized in vitro by pSFV3 expression vector and introduced into the spleen of mice, using HVJ-liposomes. A high level of beta-galactosidase activity was detected for 10 days in mouse spleen. The human me...

journal_title：Human gene therapy

authors： Zhou WZ,Hoon DS,Huang SK,Fujii S,Hashimoto K,Morishita R,Kaneda Y

更新日期：1999-11-01 00:00:00