Improved gene transfer into human lymphocytes using retroviruses with the gibbon ape leukemia virus envelope.

abstract：:The liver is an attractive target tissue for gene therapy. Current approaches for hepatic gene delivery include retroviral and adenoviral vectors, liposome/DNA, and peptide/DNA complexes. This study describes a technique for direct injection of DNA into liver that led to significant gene expression. Gene expression wa...

journal_title：Human gene therapy

authors： Hickman MA,Malone RW,Lehmann-Bruinsma K,Sih TR,Knoell D,Szoka FC,Walzem R,Carlson DM,Powell JS

更新日期：1994-12-01 00:00:00

abstract：:Adoptive cellular therapy has evolved into a powerful force in the battle against cancer, holding promise for curative responses in patients with advanced and refractory tumors. Autologous T cells, reprogrammed to target malignant cells via the expression of a chimeric antigen receptor (CAR) represent the frontrunner ...

journal_title：Human gene therapy

authors： Harrer DC,Dörrie J,Schaft N

更新日期：2018-05-01 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：: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：:The expanding use of adenoviral vectors for gene therapy has brought about the need for new analytical tools. We have developed an anion-exchange high-performance liquid chromatography method to analyze recombinant adenovirus serotype 5 samples. Before this assay, available analytical methods consisted of either long-...

journal_title：Human gene therapy

authors： Shabram PW,Giroux DD,Goudreau AM,Gregory RJ,Horn MT,Huyghe BG,Liu X,Nunnally MH,Sugarman BJ,Sutjipto S

更新日期：1997-03-01 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：:Twenty-eight patients with advanced neovascular age-related macular degeneration (AMD) were given a single intravitreous injection of an E1-, partial E3-, E4-deleted adenoviral vector expressing human pigment epithelium- derived factor (AdPEDF.11). Doses ranging from 10(6) to 10(9.5) particle units (PU) were investiga...

journal_title：Human gene therapy

authors： Campochiaro PA,Nguyen QD,Shah SM,Klein ML,Holz E,Frank RN,Saperstein DA,Gupta A,Stout JT,Macko J,DiBartolomeo R,Wei LL

更新日期：2006-02-01 00:00:00

abstract：:As efficient and less toxic virus-derived gene therapy vectors are developed, a pressing problem is to avoid immune response to the therapeutic gene product. Secreted therapeutic proteins potentially represent a special problem, as they are readily available to professional antigen-presenting cells throughout the body...

journal_title：Human gene therapy

authors： Cerullo V,McCormack W,Seiler M,Mane V,Cela R,Clarke C,Rodgers JR,Lee B

更新日期：2007-12-01 00:00:00

abstract：:At present, much more studies have focused on the role of microRNAs in osteoporosis, but the more specific role of microRNA-150-3p (miR-150-3p) in osteoporosis still needs full exploration. We aim at investigating the role of miR-150-3p in osteoporosis and at exploring the related mechanisms. Bone marrow mesenchymal s...

journal_title：Human gene therapy

authors： Qiu M,Zhai S,Fu Q,Liu D

更新日期：2021-01-22 00:00:00

abstract：:Human serum is known to inactivate many retroviruses, including murine leukemia viruses (MLV). Exposure of vectors based on MLV to human serum components would presumably decrease the efficiency of gene transfer in vivo. Human serum also lyses xenogeneic cells, which would affect the survival of retroviral vector pack...

journal_title：Human gene therapy

authors： Russell DW,Berger MS,Miller AD

更新日期：1995-05-01 00:00:00

abstract：:Low in vivo transduction efficiency and safety concerns have been hurdles for effective hematopoietic stem cell (HSC) gene therapy. Here, we investigate whether the safety and efficiency of retroviral gene transfer into HSCs can be improved by using human allogeneic umbilical cord blood (UCB)-derived supplements inste...

journal_title：Human gene therapy

authors： Moon N,Yang SJ,Park BB,Chung YS,Lee JW,Oh IH

更新日期：2008-07-01 00:00:00

abstract：:Clinical trials of gene therapy using a viral delivery system for glioma have been limited. Recently, gene therapy using stem cells as the vehicles for delivery of therapeutic agents has emerged as a new treatment strategy for malignant brain tumors. In this study, we used human umbilical cord blood-derived mesenchyma...

journal_title：Human gene therapy

authors： Ryu CH,Park SH,Park SA,Kim SM,Lim JY,Jeong CH,Yoon WS,Oh WI,Sung YC,Jeun SS

更新日期：2011-06-01 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 immunogenicity of adenovirus vectors remains a major obstacle to their safe and efficacious use for gene therapy. In order to identify T-cell epitopes directly from adenoviruses, four viral protein sequences were screened for the well-characterized 9-mer HLA-A2 binding motif. Peripheral blood mononuclear cells (PB...

journal_title：Human gene therapy

authors： Olive M,Eisenlohr L,Flomenberg N,Hsu S,Flomenberg P

更新日期：2002-07-01 00:00:00

abstract：:Deficiency of glycogen branching enzyme (GBE) causes glycogen storage disease type IV (GSD IV), which is characterized by the accumulation of a less branched, poorly soluble form of glycogen called polyglucosan (PG) in multiple tissues. This study evaluates the efficacy of gene therapy with an adeno-associated viral (...

journal_title：Human gene therapy

authors： Yi H,Zhang Q,Brooks ED,Yang C,Thurberg BL,Kishnani PS,Sun B

更新日期：2017-03-01 00:00:00

abstract：:Gene electrotransfer is gaining momentum as an efficient methodology for nonviral gene transfer. In skeletal muscle, data suggest that electric pulses play two roles: structurally permeabilizing the muscle fibers and electrophoretically supporting the migration of DNA toward or across the permeabilized membrane. To in...

journal_title：Human gene therapy

authors： André FM,Gehl J,Sersa G,Préat V,Hojman P,Eriksen J,Golzio M,Cemazar M,Pavselj N,Rols MP,Miklavcic D,Neumann E,Teissié J,Mir LM

更新日期：2008-11-01 00:00:00

abstract：:Here we show potent inhibition of HIV-1 replication in a human T cell line and primary human CD4(+) cells by expressing a single antiviral protein. Nullbasic is a mutant form of the HIV-1 Tat protein that was previously shown to strongly inhibit HIV-1 replication in nonhematopoietic cell lines by targeting three steps...

journal_title：Human gene therapy

authors： Apolloni A,Lin MH,Sivakumaran H,Li D,Kershaw MH,Harrich D

更新日期：2013-03-01 00:00:00

abstract：:Foamy virus (FV) vectors are a promising gene delivery system for use in hematopoietic stem cell gene therapy. Previous FV vector marking studies in the NOD/SCID xenotransplantation model used umbilical cord blood (UCB)-derived SCID repopulating cells (SRCs) that were assayed 5-10 weeks posttransplantation. We now rep...

journal_title：Human gene therapy

authors： Josephson NC,Trobridge G,Russell DW

更新日期：2004-01-01 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：:Serum-induced inactivation of retroviruses is the most critical limitation for in vivo gene transfer therapy. To solve this problem, we searched for reagents that protect retroviruses from inactivation. The effects of the protease inhibitors FOY-007 and FOY-305 and of an inhibitor of the complement pathway FUT-175, al...

journal_title：Human gene therapy

authors： Miyao Y,Ikenaka K,Kishima H,Tamura M,Nakamura K,Kurumi M,Hayakawa T,Shimizu K

更新日期：1997-09-01 00:00:00

abstract：:Chronic inflammation in tibialis anterior muscles of mdx mice was produced by a single injection of a recombinant adenovirus vector (AV) expressing an immunogenic beta-galactosidase (beta-gal). In regions of intense beta-gal staining, mononuclear infiltrates abounded, and muscle fibers showed strong extrasynaptic utro...

journal_title：Human gene therapy

authors： Waheed I,Gilbert R,Nalbantoglu J,Guibinga GH,Petrof BJ,Karpati G

更新日期：2005-04-01 00:00:00

abstract：:Adeno-associated viral vectors are showing great promise as gene therapy vectors for a wide range of retinal disorders. To date, evaluation of therapeutic approaches has depended almost exclusively on the use of animal models. With recent advances in human stem cell technology, stem cell-derived retina now offers the ...

journal_title：Human gene therapy

authors： Gonzalez-Cordero A,Goh D,Kruczek K,Naeem A,Fernando M,Kleine Holthaus SM,Takaaki M,Blackford SJI,Kloc M,Agundez L,Sampson RD,Borooah S,Ovando-Roche P,Mehat MS,West EL,Smith AJ,Pearson RA,Ali RR

更新日期：2018-10-01 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：:Genetically modified lymphoblastoid cell lines (LCL) have been shown to be an attractive alternative source of antigen-presenting cells for cancer vaccination in vitro. We tested their application in patients with pancreatic cancer in a phase I clinical trial. As a model tumor antigen, we selected the point-mutated (c...

journal_title：Human gene therapy

authors： Kubuschok B,Pfreundschuh M,Breit R,Hartmann F,Sester M,Gärtner B,König J,Murawski N,Held G,Zwick C,Neumann F

更新日期：2012-12-01 00:00:00

abstract：:Aerosol delivery of adenoviral vectors is of particular interest in regard to gene therapy for cystic fibrosis (CF), with potential advantages of more uniform respiratory delivery, a less invasive approach, and ease of repetition. The AdHCMVsp1LacZ (AdLacZ) adenoviral vector was used to evaluate the feasibility of aer...

journal_title：Human gene therapy

authors： Katkin JP,Gilbert BE,Langston C,French K,Beaudet AL

更新日期：1995-08-01 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：:Retinal gene therapy based on adeno-associated viral (AAV) vectors is safe and efficient in humans. The low intrinsic DNA transfer capacity of AAV has been expanded by dual vectors where a large expression cassette is split in two halves independently packaged in two AAV vectors. Dual AAV transduction efficiency, howe...

journal_title：Human gene therapy

authors： Maddalena A,Dell'Aquila F,Giovannelli P,Tiberi P,Wanderlingh LG,Montefusco S,Tornabene P,Iodice C,Visconte F,Carissimo A,Medina DL,Castoria G,Auricchio A

更新日期：2018-08-01 00:00:00

abstract：:Establishing pharmacological parameters, such as efficacy, routes of administration, and toxicity, for recombinant adeno-associated virus (rAAV) vectors is a prerequisite for gaining acceptance for clinical applications. In fact, even a therapeutic window, that is, the dose range between therapeutic efficacy and toxic...

journal_title：Human gene therapy

authors： Virag T,Cecchini S,Kotin RM

更新日期：2009-08-01 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：: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