abstract：:Vectors derived from the human parvovirus AAV-2 (adeno-associated virus type 2) are among the most promising gene delivery vehicles currently being developed. These vectors are not only capable of transducing a large variety of human cell types in vitro and in vivo, but in immunocompetent animal models can establish l...

journal_title：Human gene therapy

authors： Grimm D,Kleinschmidt JA

更新日期：1999-10-10 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：:Gene transfer for therapeutic angiogenesis represents a novel treatment for patients with chronic angina refractory to standard medical therapy and not amenable to conventional revascularization. We sought to assess the role of intraoperative multiplane transesophageal echocardiography (MPTEE) in guiding injection of ...

journal_title：Human gene therapy

authors： Esakof DD,Maysky M,Losordo DW,Vale PR,Lathi K,Pastore JO,Symes JF,Isner JM

更新日期：1999-09-20 00:00:00

abstract：:The addition of replication-defective recombinant adenovirus to plasmid transfection (termed here "adenofection") has been shown to increase plasmid transgene expression in limited studies. Similarly, the addition of cationic liposomes to adenovirus increases adenovirus-mediated gene transduction (termed here "lipoduc...

journal_title：Human gene therapy

authors： Dunphy EJ,Redman RA,Herweijer H,Cripe TP

更新日期：1999-09-20 00:00:00

abstract：:Adenovirus (Ad) vectors used for gene therapy are efficient in entering the infected cell and targeting their genome to the nucleus. To study the mechanism of the interaction between Ad and the nuclear envelope we have established an in vitro assay using rat liver nuclei incubated with serotype 5 Ad vector. Binding of...

journal_title：Human gene therapy

authors： Wisnivesky JP,Leopold PL,Crystal RG

更新日期：1999-09-01 00:00:00

abstract：:The influence of serum on the production of retroviral vectors by the HT1080 human fibrosarcoma-derived packaging cell line FLYRD18 was investigated. A fourfold increase in virus titer was observed under serum-free conditions, as compared with medium supplemented with 10% fetal calf serum. A similar improvement was al...

journal_title：Human gene therapy

authors： Gerin PA,Gilligan MG,Searle PF,Al-Rubeai M

更新日期：1999-08-10 00:00:00

abstract：:CTL lines directed against HIV-1 antigens were generated from infected individuals and were transduced by the HMB-K(b)HuIFNbeta vector, resulting in low, constitutive expression of interferon beta (IFN-beta). The IFN-beta-transduced cells showed markedly increased HIV-1-specific, MHC class I-restricted CTL activity ag...

journal_title：Human gene therapy

authors： Hadida F,De Maeyer E,Cremer I,Autran B,Baggiolini M,Debré P,Vieillard V

更新日期：1999-07-20 00:00:00

abstract：:La Crosse virus (LACV)-mediated encephalitis is the most frequently reported arboviral disease in the United States, but to date no vaccine against this virus is available. We have established a new animal model, genetically targeted mice lacking a functional interferon type I receptor (IFNAR-1). These mice show an ag...

journal_title：Human gene therapy

authors： Schuh T,Schultz J,Moelling K,Pavlovic J

更新日期：1999-07-01 00:00:00

abstract：:Hematopoietic stem cells (HSCs) are a potential target for the retrovirus-mediated transfer of chemotherapeutic drug resistance genes. For integration of the proviral DNA in the HSC genome cell division is required. In the bone marrow (BM) hematopoiesis occurs in the vicinity of stroma cells. Soluble stroma components...

journal_title：Human gene therapy

authors： Schiedlmeier B,Buss EC,Veldwijk MR,Zeller WJ,Fruehauf S

更新日期：1999-06-10 00:00:00

abstract：:The purpose of this study was to determine the safety and antitumor activity of IFN-gamma retroviral vector in patients with advanced melanoma. Seventeen patients (9 single courses, 8 multiple courses) received a total of 363 intratumor injections of IFN-gamma retroviral vector (1 x 10(7) PFU/ml administered at 0.3, 0...

journal_title：Human gene therapy

authors： Nemunaitis J,Fong T,Burrows F,Bruce J,Peters G,Ognoskie N,Meyer W,Wynne D,Kerr R,Pippen J,Oldham F,Ando D

更新日期：1999-05-20 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：:Replication-deficient adenoviruses are known to induce acute injury and inflammation of infected tissues, thus limiting their use for human gene therapy. However, molecular mechanisms triggering this response have not been fully defined. To characterize this response, chemokine expression was evaluated in DBA/2 mice f...

journal_title：Human gene therapy

authors： Muruve DA,Barnes MJ,Stillman IE,Libermann TA

更新日期：1999-04-10 00:00:00

abstract：:Administration of plasmid/lipid complexes to the lung airways for the treatment of metastatic pulmonary diseases represents a new strategy of gene therapy. In this study we present evidence that intratracheal administration of a plasmid encoding murine IL-12 complexed with N-[1-(2,3-dioleyloxy)propyl)-N,N,N-trimethyla...

journal_title：Human gene therapy

authors： Blezinger P,Freimark BD,Matar M,Wilson E,Singhal A,Min W,Nordstrom JL,Pericle F

更新日期：1999-03-20 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：:We studied hematopoietic progenitors from fetal baboon blood, marrow, and liver at four time points (125, 140, 160, and 175 days) during the third trimester (gestation approximately 180 days) to determine if fetal baboons might be an appropriate model for in utero gene therapy of hematopoietic stem cells (HSCs). Cells...

journal_title：Human gene therapy

authors： Winkler A,Kiem HP,Shields LE,Sun QH,Andrews RG

更新日期：1999-03-01 00:00:00

abstract：:Cell encapsulation offers a safe and manufacturable method for the systemic delivery of therapeutic proteins from genetically engineered cells. However, control of dose delivery remains a major issue with regard to clinical application. We generated populations of immortalized murine NIH 3T3 fibroblasts that secrete m...

journal_title：Human gene therapy

authors： Serguera C,Bohl D,Rolland E,Prevost P,Heard JM

更新日期：1999-02-10 00:00:00

abstract：:Adenovirus vectors transduce liver hepatocytes with extreme efficiency; however, transgene expression is eliminated within 2 weeks. Extinction of transgene expression has been attributed to infiltrating cytotoxic T lymphocytes (CTLs) in the liver in a process that resembles a number of human diseases, including viral ...

journal_title：Human gene therapy

authors： Chirmule N,Moscioni AD,Qian Y,Qian R,Chen Y,Wilson JM

更新日期：1999-01-20 00:00:00

abstract：:Somatic gene therapy using nonautologous recombinant cells immunologically protected with alginate microcapsules has been successfully used to treat rodent genetic diseases. We now report the delivery of recombinant gene products to the brain in rodents by implanting microencapsulated cells for the purpose of eventual...

journal_title：Human gene therapy

authors： Ross CJ,Ralph M,Chang PL

更新日期：1999-01-01 00:00:00

abstract：:Autoimmune destruction of islets in the pancreas leads to the development of insulin-dependent diabetes mellitus (IDDM). Replacement of insulin-producing tissue by transplantation of islets provides a cure to disease but requires immunosuppression or a means of controlling anti-graft immune responses. To promote islet...

journal_title：Human gene therapy

authors： Gallichan WS,Kafri T,Krahl T,Verma IM,Sarvetnick N

更新日期：1998-12-10 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：:Malignant mesothelioma is a tumor of the pleura for which there is no satisfactory treatment. It is almost universally fatal, regardless of the stage of the tumor at the time of diagnosis. Current treatment modalities include surgery, chemotherapy, and radiation therapy, although in some series none of these modalitie...

journal_title：Human gene therapy

authors： Schwarzenberger P,Harrison L,Weinacker A,Marrogi A,Byrne P,Ramesh R,Theodossiou C,Gaumer R,Summer W,Freeman SM,Kolls JK

更新日期：1998-11-20 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：:This Phase I study, "Ribozyme Gene Therapy of HIV-1 Infection" (UCSD HSC #971072, FDA BB-IND 6405), is a prospective, open-label trial of infusion of autologous gene-altered cells into asymptomatic HIV-1 seropositive individuals. The objectives of this trial are to test the safety, feasibility, and potential efficacy ...

journal_title：Human gene therapy

authors： Wong-Staal F,Poeschla EM,Looney DJ

更新日期：1998-11-01 00:00:00

abstract：:Cotransfer of a therapeutic gene together with the human MDR1 gene provides an opportunity to increase the number of transduced marrow cells, expressing the therapeutic gene, by in vivo selection for MDR1. We have used an Lg-MDR1-IRES-neo (LgMIN) retroviral vector, containing MDR1 and neo genes, separated by the EMCV ...

journal_title：Human gene therapy

authors： Zaboikin MM,Schuening FG

更新日期：1998-10-10 00:00:00

abstract：:The purpose of this study was to assess the therapeutic potential of injecting the gene for HLA-B7/beta2-microglobulin into the subcutaneous metastatic nodules of patients who are refractory to conventional treatments. The nine patients evaluated were divided into three groups and given escalating doses of DNA (20, 40...

journal_title：Human gene therapy

authors： Heo DS,Yoon SJ,Kim WS,Lee KH,Seol JG,Lee SG,Jung CW,Cho EK,Kim CW,Park MH,Sung MW,Kim KH,Bang YJ,Kim NK

更新日期：1998-09-20 00:00:00

abstract：:Adenoviruses are attractive vectors for gene transfer into cardiac muscle. However, their promiscuous tissue tropism, which leads to an ectopic expression of the transgene, is a considerable limitation. To restrict expression to cardiomyocytes, we have constructed two recombinant adenoviruses (Ad-MLC2-250betagal and A...

journal_title：Human gene therapy

authors： Griscelli F,Gilardi-Hebenstreit P,Hanania N,Franz WM,Opolon P,Perricaudet M,Ragot T

更新日期：1998-09-01 00:00:00

abstract：:We previously demonstrated that intramuscular plasmid injection serves as a useful method of long-term systemic delivery of cytokines. In the present study, we assess intramuscular DNA injection as a means of systemically delivering interleukin 10 (IL-10), a cytokine with immunosuppressive properties, and preventing t...

journal_title：Human gene therapy

authors： Nitta Y,Tashiro F,Tokui M,Shimada A,Takei I,Tabayashi K,Miyazaki J

更新日期：1998-08-10 00:00:00

abstract：:Advanced prostate cancer is invariably lethal once it becomes androgen independent (AI). With the aim of developing a new treatment we have used the human androgen-independent prostate cancer cell line, PC-3, to evaluate the effectiveness of two enzyme-directed prodrug therapy (EPT) systems as a novel means for promot...

journal_title：Human gene therapy

authors： Martiniello-Wilks R,Garcia-Aragon J,Daja MM,Russell P,Both GW,Molloy PL,Lockett LJ,Russell PJ

更新日期：1998-07-20 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：:Laminin-5 is composed of three distinct polypeptides, alpha3, beta3, and gamma2, which are encoded by three different genes, LAMA3, LAMB3, and LAMC2, respectively. We have isolated epidermal keratinocytes from a patient presenting with a lethal form of junctional epidermolysis bullosa characterized by a homozygous mut...

journal_title：Human gene therapy

authors： Dellambra E,Vailly J,Pellegrini G,Bondanza S,Golisano O,Macchia C,Zambruno G,Meneguzzi G,De Luca M

更新日期：1998-06-10 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：:Herpes simplex virus thymidine kinase (HSV-tk) gene therapy for brain tumors depends on ganciclovir (GCV) and its transport across the blood-brain tumor barrier (BBTB). We examined whether RMP-7, the bradykinin analog and potent BBTB permeabilizer, could enhance the efficacy of GCV treatment of brain tumors by increas...

journal_title：Human gene therapy

authors： LeMay DR,Kittaka M,Gordon EM,Gray B,Stins MF,McComb JG,Jovanovic S,Tabrizi P,Weiss MH,Bartus R,Anderson WF,Zlokovic BV

更新日期：1998-05-01 00:00:00

abstract：:As an alternative to virus-mediated gene transfer, we previously demonstrated a simple, safe, and efficient transfer of foreign gene into the central nervous system using continuous injection of a plasmid DNA-cationic liposome complex. To explore whether this approach can be applied to the treatment of certain neurolo...

journal_title：Human gene therapy

authors： Imaoka T,Date I,Ohmoto T,Nagatsu T

更新日期：1998-05-01 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：:Immunoisolation of allogeneic cells within a membrane-bound device is a unique approach for gene therapy. We employed an immunoisolation device that protects allograft, but not xenograft, cells from destruction, to implant a human fibroblast line (MSU 1.2) in athymic rodents. Cells, transduced with the MFG-human facto...

journal_title：Human gene therapy

authors： Brauker J,Frost GH,Dwarki V,Nijjar T,Chin R,Carr-Brendel V,Jasunas C,Hodgett D,Stone W,Cohen LK,Johnson RC

更新日期：1998-04-10 00:00:00

abstract：:Human immunodeficiency virus (HIV) infection represents one of the most challenging systems for gene therapy. Thanks to the extended knowledge of the molecular biology of the HIV life cycle, many different strategies have been developed including transdominant modifications of HIV proteins, RNA decoys, antisense RNA, ...

journal_title：Human gene therapy

authors： Michienzi A,Conti L,Varano B,Prislei S,Gessani S,Bozzoni I

更新日期：1998-03-20 00:00:00

abstract：:Previously, we described a nonviral cytoplasmic gene therapy vector system based on the T7 autogene concept. This system has been shown to achieve rapid and high levels of gene expression in a variety of animal cells and tissues. To test the utility of the system in vivo tumor ablation, a T7 cancer gene therapy plasmi...

journal_title：Human gene therapy

authors： Chen X,Li Y,Xiong K,Aizicovici S,Xie Y,Zhu Q,Sturtz F,Shulok J,Snodgrass R,Wagner TE,Platika D

更新日期：1998-03-20 00:00:00

abstract：:Current clinical gene therapy protocols for the treatment of human immunodeficiency virus type 1 (HIV-1) infection often involve the ex vivo transduction and expansion of CD4+ T cells derived from HIV-positive patients at a late stage in their disease (CD4 count <400). These protocols involve the transduction of T cel...

journal_title：Human gene therapy

authors： Poznansky MC,Foxall R,Mhashilkar A,Coker R,Jones S,Ramstedt U,Marasco W

更新日期：1998-03-01 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：:A novel retroviral vector has been designed based on a Friend-murine leukemia virus (Fr-MuLV) FB29 strain. The latter has been selected according to characteristics of pathogenicity in mice where it induces a disease of the haemopoietic system affecting all lineages. Higher infectivity has also been demonstrated as co...

journal_title：Human gene therapy

authors： Cohen-Haguenauer O,Restrepo LM,Masset M,Bayer J,Dal Cortivo L,Marolleau JP,Benbunan M,Boiron M,Marty M

更新日期：1998-01-20 00:00:00