Constraints on Human CD34+ Cell Fate due to Lentiviral Vectors Can Be Relieved by Valproic Acid.

abstract：:Human embryonic stem cells (hESC) and induced pluripotent stem cells (iPSC) offer great hope for in vitro modeling of Parkinson's disease (PD), as well as for designing cell-replacement therapies. To realize these opportunities, there is an urgent need to develop efficient protocols for the directed differentiation of...

journal_title：Human gene therapy

authors： Sánchez-Danés A,Consiglio A,Richaud Y,Rodríguez-Pizà I,Dehay B,Edel M,Bové J,Memo M,Vila M,Raya A,Izpisua Belmonte JC

更新日期：2012-01-01 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

abstract：:Efficient DNA electrotransfer can be achieved with combinations of short high-voltage (HV) and long low voltage (LV) pulses that cover two effects of the pulses, namely, target cell electropermeabilization and DNA electrophoresis within the tissue. Because HV and LV can be delivered with a lag up to 3000 sec between t...

journal_title：Human gene therapy

authors： Satkauskas S,André F,Bureau MF,Scherman D,Miklavcic D,Mir LM

更新日期：2005-10-01 00:00:00

abstract：:Gene therapy for hemophilia B has been shown to result in long-term expression and immune tolerance to factor IX (F.IX) after in vivo transduction of hepatocytes with adeno-associated viral (AAV-2) vectors in experimental animals. An optimized protocol was effective in several strains of mice with a factor 9 gene dele...

journal_title：Human gene therapy

authors： Cooper M,Nayak S,Hoffman BE,Terhorst C,Cao O,Herzog RW

更新日期：2009-07-01 00:00:00

abstract：:Spinal muscular atrophy (SMA) is an autosomal recessive disease affecting ∼1 in 10,000 live births. The most striking component is the loss of α-motor neurons in the ventral horn of the spinal cord, resulting in progressive paralysis and eventually premature death. There is no current treatment paradigm other than sup...

journal_title：Human gene therapy

authors： Porensky PN,Burghes AH

更新日期：2013-05-01 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：:Classical gene therapy for cystic fibrosis has had limited success because of immune response against viral vectors and short-term expression of cDNA-based transgenes. These limitations could be overcome by delivering the complete genomic CFTR gene on nonintegrating human artificial chromosomes (HACs). Here, we report...

journal_title：Human gene therapy

authors： Rocchi L,Braz C,Cattani S,Ramalho A,Christan S,Edlinger M,Ascenzioni F,Laner A,Kraner S,Amaral M,Schindelhauer D

更新日期：2010-09-01 00:00:00

abstract：:Adenovirus-polylysine-DNA complexes were evaluated for their capacity to accomplish direct in vivo gene transfer to airway epithelium employing a rodent model. Binary complexes containing transferrin or adenovirus, or combination complexes containing both transferrin and adenovirus, were evaluated. The highest in vitr...

journal_title：Human gene therapy

authors： Gao L,Wagner E,Cotten M,Agarwal S,Harris C,Rømer M,Miller L,Hu PC,Curiel D

更新日期：1993-02-01 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：: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：: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：: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：: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：:Successful gene therapy approaches for metachromatic leukodystrophy (MLD), based either on hematopoietic stem/progenitor cells (HSPCs) or direct central nervous system (CNS) gene transfer, highlighted a requirement for high levels of arylsulfatase A (ARSA) expression to achieve correction of disease manifestations in ...

journal_title：Human gene therapy

authors： Capotondo A,Cesani M,Pepe S,Fasano S,Gregori S,Tononi L,Venneri MA,Brambilla R,Quattrini A,Ballabio A,Cosma MP,Naldini L,Biffi A

更新日期：2007-09-01 00:00:00

abstract：:In summary, I will reiterate the five points I would like to leave with you today: First, the biological revolution has extraordinary power to do good. As long as the use of our new genetic knowledge is guided by the traditional ideals of the healing professions--to help improve the human condition without doing harm-...

journal_title：Human gene therapy

authors： Healy B

更新日期：1992-02-01 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：:Adrenomedullin (AM) has been shown to protect against ischemia/reperfusion-induced myocardial infarction and apoptosis. In the present study, we examined the potential neuroprotective action of delayed AM gene transfer in cerebral ischemia. Three days after a 1-hr occlusion of the middle cerebral artery (MCAO), rats w...

journal_title：Human gene therapy

authors： Xia CF,Yin H,Borlongan CV,Chao J,Chao L

更新日期：2004-12-01 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：:Expression of a gene encoding the diphtheria toxin A (DT-A) fragment, controlled by tissue specific regulatory elements, has previously been used to kill selected cell populations. Here, we have examined the feasibility of controlling DT-A expression using regulatory systems from the human immunodeficiency virus (HIV-...

journal_title：Human gene therapy

authors： Harrison GS,Maxwell F,Long CJ,Rosen CA,Glode LM,Maxwell IH

更新日期：1991-04-01 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：: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：: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：:An alternative form of gene therapy involves immunoisolation of a nonautologous cell line engineered to secrete a therapeutic product. Encapsulation of these cells in a biocompatible polymer serves to protect these allogeneic cells from host-versus-graft rejection while recombinant products and nutrients are able to p...

journal_title：Human gene therapy

authors： Cirone P,Bourgeois JM,Shen F,Chang PL

更新日期：2004-10-01 00:00:00

abstract：:Mutations in the gene encoding the peroxisomal ATP-binding cassette transporter (ABCD1) cause elevations in very long-chain fatty acids (VLCFAs) and the neurodegenerative disease adrenoleukodystrophy (ALD). In most adults, this manifests as the spinal cord axonopathy adrenomyeloneuropathy (AMN). A challenge in virus-b...

journal_title：Human gene therapy

authors： Gong Y,Berenson A,Laheji F,Gao G,Wang D,Ng C,Volak A,Kok R,Kreouzis V,Dijkstra IM,Kemp S,Maguire CA,Eichler F

更新日期：2019-05-01 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：: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：: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：: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：:Traditionally, skeletal muscle and liver are the preferred target organs for gene transfer to supply a transgene product into the systemic circulation. In this respect, adipose tissue presents a number of attractive features. However, adipose tissue transduction in vivo has not been feasible by conventional methods. T...

journal_title：Human gene therapy

authors： Mizukami H,Mimuro J,Ogura T,Okada T,Urabe M,Kume A,Sakata Y,Ozawa K

更新日期：2006-09-01 00:00:00

abstract：:Adenovirus type 5 (Ad5) is a commonly used vector for gene therapy, but its efficacy is limited by high seroprevalence and off-target hepatic and splenic sequestration. In order to circumvent these limitations, the use of vectors derived from rare species adenoviruses is appealing. The opportunity to retarget rare spe...

journal_title：Human gene therapy

authors： Coughlan L,Uusi-Kerttula H,Ma J,Degg BP,Parker AL,Baker AH

更新日期：2014-04-01 00:00:00